Genetic Therapy: Techniques and strategies which include the use of coding sequences and other conventional or radical means to transform or modify cells for the purpose of treating or reversing disease conditions.Genetic Vectors: DNA molecules capable of autonomous replication within a host cell and into which other DNA sequences can be inserted and thus amplified. Many are derived from PLASMIDS; BACTERIOPHAGES; or VIRUSES. They are used for transporting foreign genes into recipient cells. Genetic vectors possess a functional replicator site and contain GENETIC MARKERS to facilitate their selective recognition.Gene Transfer Techniques: The introduction of functional (usually cloned) GENES into cells. A variety of techniques and naturally occurring processes are used for the gene transfer such as cell hybridization, LIPOSOMES or microcell-mediated gene transfer, ELECTROPORATION, chromosome-mediated gene transfer, TRANSFECTION, and GENETIC TRANSDUCTION. Gene transfer may result in genetically transformed cells and individual organisms.Transduction, Genetic: The transfer of bacterial DNA by phages from an infected bacterium to another bacterium. This also refers to the transfer of genes into eukaryotic cells by viruses. This naturally occurring process is routinely employed as a GENE TRANSFER TECHNIQUE.Adenoviridae: A family of non-enveloped viruses infecting mammals (MASTADENOVIRUS) and birds (AVIADENOVIRUS) or both (ATADENOVIRUS). Infections may be asymptomatic or result in a variety of diseases.Dependovirus: A genus of the family PARVOVIRIDAE, subfamily PARVOVIRINAE, which are dependent on a coinfection with helper adenoviruses or herpesviruses for their efficient replication. The type species is Adeno-associated virus 2.Transfection: The uptake of naked or purified DNA by CELLS, usually meaning the process as it occurs in eukaryotic cells. It is analogous to bacterial transformation (TRANSFORMATION, BACTERIAL) and both are routinely employed in GENE TRANSFER TECHNIQUES.Drug Delivery Systems: Systems for the delivery of drugs to target sites of pharmacological actions. Technologies employed include those concerning drug preparation, route of administration, site targeting, metabolism, and toxicity.Pharmaceutical Vehicles: A carrier or inert medium used as a solvent (or diluent) in which the medicinally active agent is formulated and or administered. (Dictionary of Pharmacy, 1986)Plasmids: Extrachromosomal, usually CIRCULAR DNA molecules that are self-replicating and transferable from one organism to another. They are found in a variety of bacterial, archaeal, fungal, algal, and plant species. They are used in GENETIC ENGINEERING as CLONING VECTORS.Drug Carriers: Forms to which substances are incorporated to improve the delivery and the effectiveness of drugs. Drug carriers are used in drug-delivery systems such as the controlled-release technology to prolong in vivo drug actions, decrease drug metabolism, and reduce drug toxicity. Carriers are also used in designs to increase the effectiveness of drug delivery to the target sites of pharmacological actions. Liposomes, albumin microspheres, soluble synthetic polymers, DNA complexes, protein-drug conjugates, and carrier erythrocytes among others have been employed as biodegradable drug carriers.Nanoparticles: Nanometer-sized particles that are nanoscale in three dimensions. They include nanocrystaline materials; NANOCAPSULES; METAL NANOPARTICLES; DENDRIMERS, and QUANTUM DOTS. The uses of nanoparticles include DRUG DELIVERY SYSTEMS and cancer targeting and imaging.Polyethyleneimine: Strongly cationic polymer that binds to certain proteins; used as a marker in immunology, to precipitate and purify enzymes and lipids. Synonyms: aziridine polymer; Epamine; Epomine; ethylenimine polymer; Montrek; PEI; Polymin(e).Genes, Viral: The functional hereditary units of VIRUSES.Lentivirus: A genus of the family RETROVIRIDAE consisting of non-oncogenic retroviruses that produce multi-organ diseases characterized by long incubation periods and persistent infection. Lentiviruses are unique in that they contain open reading frames (ORFs) between the pol and env genes and in the 3' env region. Five serogroups are recognized, reflecting the mammalian hosts with which they are associated. HIV-1 is the type species.Transgenes: Genes that are introduced into an organism using GENE TRANSFER TECHNIQUES.Liposomes: Artificial, single or multilaminar vesicles (made from lecithins or other lipids) that are used for the delivery of a variety of biological molecules or molecular complexes to cells, for example, drug delivery and gene transfer. They are also used to study membranes and membrane proteins.Gene Expression Regulation, Viral: Any of the processes by which cytoplasmic factors influence the differential control of gene action in viruses.Polymers: Compounds formed by the joining of smaller, usually repeating, units linked by covalent bonds. These compounds often form large macromolecules (e.g., BIOPOLYMERS; PLASTICS).Gene Expression: The phenotypic manifestation of a gene or genes by the processes of GENETIC TRANSCRIPTION and GENETIC TRANSLATION.Retroviridae: Family of RNA viruses that infects birds and mammals and encodes the enzyme reverse transcriptase. The family contains seven genera: DELTARETROVIRUS; LENTIVIRUS; RETROVIRUSES TYPE B, MAMMALIAN; ALPHARETROVIRUS; GAMMARETROVIRUS; RETROVIRUSES TYPE D; and SPUMAVIRUS. A key feature of retrovirus biology is the synthesis of a DNA copy of the genome which is integrated into cellular DNA. After integration it is sometimes not expressed but maintained in a latent state (PROVIRUSES).Green Fluorescent Proteins: Protein analogs and derivatives of the Aequorea victoria green fluorescent protein that emit light (FLUORESCENCE) when excited with ULTRAVIOLET RAYS. They are used in REPORTER GENES in doing GENETIC TECHNIQUES. Numerous mutants have been made to emit other colors or be sensitive to pH.Gene Targeting: The integration of exogenous DNA into the genome of an organism at sites where its expression can be suitably controlled. This integration occurs as a result of homologous recombination.Cell Line: Established cell cultures that have the potential to propagate indefinitely.Polylysine: A peptide which is a homopolymer of lysine.Particle Size: Relating to the size of solids.Genes, Transgenic, Suicide: Genes that are used transgenically, i.e., via GENE TRANSFER TECHNIQUES to induce CELL DEATH.Thymidine Kinase: An enzyme that catalyzes the conversion of ATP and thymidine to ADP and thymidine 5'-phosphate. Deoxyuridine can also act as an acceptor and dGTP as a donor. (From Enzyme Nomenclature, 1992) EC Nanometer-sized, hollow, spherically-shaped objects that can be utilized to encapsulate small amounts of pharmaceuticals, enzymes, or other catalysts (Glossary of Biotechnology and Nanobiotechnology, 4th ed).Polyglycolic Acid: A biocompatible polymer used as a surgical suture material.Microbubbles: Small encapsulated gas bubbles (diameters of micrometers) that can be used as CONTRAST MEDIA, and in other diagnostic and therapeutic applications. Upon exposure to sufficiently intense ultrasound, microbubbles will cavitate, rupture, disappear, release gas content. Such characteristics of the microbubbles can be used to enhance diagnostic tests, dissolve blood clots, and deliver drugs or genes for therapy.Cell Line, Tumor: A cell line derived from cultured tumor cells.beta-Galactosidase: A group of enzymes that catalyzes the hydrolysis of terminal, non-reducing beta-D-galactose residues in beta-galactosides. Deficiency of beta-Galactosidase A1 may cause GANGLIOSIDOSIS, GM1.Mice, Nude: Mutant mice homozygous for the recessive gene "nude" which fail to develop a thymus. They are useful in tumor studies and studies on immune responses.DNA: A deoxyribonucleotide polymer that is the primary genetic material of all cells. Eukaryotic and prokaryotic organisms normally contain DNA in a double-stranded state, yet several important biological processes transiently involve single-stranded regions. DNA, which consists of a polysugar-phosphate backbone possessing projections of purines (adenine and guanine) and pyrimidines (thymine and cytosine), forms a double helix that is held together by hydrogen bonds between these purines and pyrimidines (adenine to thymine and guanine to cytosine).Disease Models, Animal: Naturally occurring or experimentally induced animal diseases with pathological processes sufficiently similar to those of human diseases. They are used as study models for human diseases.Electroporation: A technique in which electric pulses of intensity in kilovolts per centimeter and of microsecond-to-millisecond duration cause a temporary loss of the semipermeability of CELL MEMBRANES, thus leading to ion leakage, escape of metabolites, and increased uptake by cells of drugs, molecular probes, and DNA.Chitosan: Deacetylated CHITIN, a linear polysaccharide of deacetylated beta-1,4-D-glucosamine. It is used in HYDROGEL and to treat WOUNDS.Polyethylene Glycols: Polymers of ETHYLENE OXIDE and water, and their ethers. They vary in consistency from liquid to solid depending on the molecular weight indicated by a number following the name. They are used as SURFACTANTS, dispersing agents, solvents, ointment and suppository bases, vehicles, and tablet excipients. Some specific groups are NONOXYNOLS, OCTOXYNOLS, and POLOXAMERS.Ganciclovir: An ACYCLOVIR analog that is a potent inhibitor of the Herpesvirus family including cytomegalovirus. Ganciclovir is used to treat complications from AIDS-associated cytomegalovirus infections.Prodrugs: A compound that, on administration, must undergo chemical conversion by metabolic processes before becoming the pharmacologically active drug for which it is a prodrug.Genetic Engineering: Directed modification of the gene complement of a living organism by such techniques as altering the DNA, substituting genetic material by means of a virus, transplanting whole nuclei, transplanting cell hybrids, etc.Mice, Inbred C57BLMice, Inbred BALB CDelivery, Obstetric: Delivery of the FETUS and PLACENTA under the care of an obstetrician or a health worker. Obstetric deliveries may involve physical, psychological, medical, or surgical interventions.Ultrasonics: A subfield of acoustics dealing in the radio frequency range higher than acoustic SOUND waves (approximately above 20 kilohertz). Ultrasonic radiation is used therapeutically (DIATHERMY and ULTRASONIC THERAPY) to generate HEAT and to selectively destroy tissues. It is also used in diagnostics, for example, ULTRASONOGRAPHY; ECHOENCEPHALOGRAPHY; and ECHOCARDIOGRAPHY, to visually display echoes received from irradiated tissues.Nanostructures: Materials which have structured components with at least one dimension in the range of 1 to 100 nanometers. These include NANOCOMPOSITES; NANOPARTICLES; NANOTUBES; and NANOWIRES.Cations: Positively charged atoms, radicals or groups of atoms which travel to the cathode or negative pole during electrolysis.Cells, Cultured: Cells propagated in vitro in special media conducive to their growth. Cultured cells are used to study developmental, morphologic, metabolic, physiologic, and genetic processes, among others.Tumor Cells, Cultured: Cells grown in vitro from neoplastic tissue. If they can be established as a TUMOR CELL LINE, they can be propagated in cell culture indefinitely.Nanomedicine: The branch of medicine concerned with the application of NANOTECHNOLOGY to the prevention and treatment of disease. It involves the monitoring, repair, construction, and control of human biological systems at the molecular level, using engineered nanodevices and NANOSTRUCTURES. (From Freitas Jr., Nanomedicine, vol 1, 1999).Biocompatible Materials: Synthetic or natural materials, other than DRUGS, that are used to replace or repair any body TISSUES or bodily function.Genes, Reporter: Genes whose expression is easily detectable and therefore used to study promoter activity at many positions in a target genome. In recombinant DNA technology, these genes may be attached to a promoter region of interest.Hydrogels: Water swollen, rigid, 3-dimensional network of cross-linked, hydrophilic macromolecules, 20-95% water. They are used in paints, printing inks, foodstuffs, pharmaceuticals, and cosmetics. (Grant & Hackh's Chemical Dictionary, 5th ed)Dendrimers: Tree-like, highly branched, polymeric compounds. They grow three-dimensionally by the addition of shells of branched molecules to a central core. The overall globular shape and presence of cavities gives potential as drug carriers and CONTRAST AGENTS.Virus Replication: The process of intracellular viral multiplication, consisting of the synthesis of PROTEINS; NUCLEIC ACIDS; and sometimes LIPIDS, and their assembly into a new infectious particle.Nanoconjugates: Tailored macromolecules harboring covalently-bound biologically active modules that target specific tissues and cells. The active modules or functional groups can include drugs, prodrugs, antibodies, and oligonucleotides, which can act synergistically and be multitargeting.Luciferases: Enzymes that oxidize certain LUMINESCENT AGENTS to emit light (PHYSICAL LUMINESCENCE). The luciferases from different organisms have evolved differently so have different structures and substrates.Cytosine Deaminase: An enzyme which catalyzes the deamination of CYTOSINE resulting in the formation of URACIL. It can also act on 5-methylcytosine to form THYMIDINE.Adenoviruses, Human: Species of the genus MASTADENOVIRUS, causing a wide range of diseases in humans. Infections are mostly asymptomatic, but can be associated with diseases of the respiratory, ocular, and gastrointestinal systems. Serotypes (named with Arabic numbers) have been grouped into species designated Human adenovirus A-F.Motor Vehicles: AUTOMOBILES, trucks, buses, or similar engine-driven conveyances. (From Random House Unabridged Dictionary, 2d ed)Cell Survival: The span of viability of a cell characterized by the capacity to perform certain functions such as metabolism, growth, reproduction, some form of responsiveness, and adaptability.Viral Proteins: Proteins found in any species of virus.Luminescent Proteins: Proteins which are involved in the phenomenon of light emission in living systems. Included are the "enzymatic" and "non-enzymatic" types of system with or without the presence of oxygen or co-factors.HeLa Cells: The first continuously cultured human malignant CELL LINE, derived from the cervical carcinoma of Henrietta Lacks. These cells are used for VIRUS CULTIVATION and antitumor drug screening assays.Nanotechnology: The development and use of techniques to study physical phenomena and construct structures in the nanoscale size range or smaller.Promoter Regions, Genetic: DNA sequences which are recognized (directly or indirectly) and bound by a DNA-dependent RNA polymerase during the initiation of transcription. Highly conserved sequences within the promoter include the Pribnow box in bacteria and the TATA BOX in eukaryotes.Cell-Penetrating Peptides: Peptides that have the ability to enter cells by crossing the plasma membrane directly, or through uptake by the endocytotic pathway.Administration, Ophthalmic: Application of pharmaceutically active agents on the tissues of the EYE.Microspheres: Small uniformly-sized spherical particles, of micrometer dimensions, frequently labeled with radioisotopes or various reagents acting as tags or markers.Coxsackie and Adenovirus Receptor-Like Membrane Protein: An Ig superfamily transmembrane protein that localizes to junctional complexes that occur between ENDOTHELIAL CELLS and EPTHELIAL CELLS. The protein may play a role in cell-cell adhesion and is the primary site for the attachment of ADENOVIRUSES during infection.Molecular Sequence Data: Descriptions of specific amino acid, carbohydrate, or nucleotide sequences which have appeared in the published literature and/or are deposited in and maintained by databanks such as GENBANK, European Molecular Biology Laboratory (EMBL), National Biomedical Research Foundation (NBRF), or other sequence repositories.Time Factors: Elements of limited time intervals, contributing to particular results or situations.Neoplasms: New abnormal growth of tissue. Malignant neoplasms show a greater degree of anaplasia and have the properties of invasion and metastasis, compared to benign neoplasms.Recombinant Fusion Proteins: Recombinant proteins produced by the GENETIC TRANSLATION of fused genes formed by the combination of NUCLEIC ACID REGULATORY SEQUENCES of one or more genes with the protein coding sequences of one or more genes.Lac Operon: The genetic unit consisting of three structural genes, an operator and a regulatory gene. The regulatory gene controls the synthesis of the three structural genes: BETA-GALACTOSIDASE and beta-galactoside permease (involved with the metabolism of lactose), and beta-thiogalactoside acetyltransferase.RNA, Small Interfering: Small double-stranded, non-protein coding RNAs (21-31 nucleotides) involved in GENE SILENCING functions, especially RNA INTERFERENCE (RNAi). Endogenously, siRNAs are generated from dsRNAs (RNA, DOUBLE-STRANDED) by the same ribonuclease, Dicer, that generates miRNAs (MICRORNAS). The perfect match of the siRNAs' antisense strand to their target RNAs mediates RNAi by siRNA-guided RNA cleavage. siRNAs fall into different classes including trans-acting siRNA (tasiRNA), repeat-associated RNA (rasiRNA), small-scan RNA (scnRNA), and Piwi protein-interacting RNA (piRNA) and have different specific gene silencing functions.Viruses: Minute infectious agents whose genomes are composed of DNA or RNA, but not both. They are characterized by a lack of independent metabolism and the inability to replicate outside living host cells.Mesenchymal Stromal Cells: Bone-marrow-derived, non-hematopoietic cells that support HEMATOPOETIC STEM CELLS. They have also been isolated from other organs and tissues such as UMBILICAL CORD BLOOD, umbilical vein subendothelium, and WHARTON JELLY. These cells are considered to be a source of multipotent stem cells because they include subpopulations of mesenchymal stem cells.Cation Exchange Resins: High molecular weight insoluble polymers which contain functional anionic groups that are capable of undergoing exchange reactions with cations.Simplexvirus: A genus of the family HERPESVIRIDAE, subfamily ALPHAHERPESVIRINAE, consisting of herpes simplex-like viruses. The type species is HERPESVIRUS 1, HUMAN.Rats, Sprague-Dawley: A strain of albino rat used widely for experimental purposes because of its calmness and ease of handling. It was developed by the Sprague-Dawley Animal Company.Base Sequence: The sequence of PURINES and PYRIMIDINES in nucleic acids and polynucleotides. It is also called nucleotide sequence.Xenograft Model Antitumor Assays: In vivo methods of screening investigative anticancer drugs, biologic response modifiers or radiotherapies. Human tumor tissue or cells are transplanted into mice or rats followed by tumor treatment regimens. A variety of outcomes are monitored to assess antitumor effectiveness.X-Linked Combined Immunodeficiency Diseases: Forms of combined immunodeficiency caused by mutations in the gene for INTERLEUKIN RECEPTOR COMMON GAMMA SUBUNIT. Both severe and non-severe subtypes of the disease have been identified.Delayed-Action Preparations: Dosage forms of a drug that act over a period of time by controlled-release processes or technology.Recombinant Proteins: Proteins prepared by recombinant DNA technology.Herpesvirus 1, Human: The type species of SIMPLEXVIRUS causing most forms of non-genital herpes simplex in humans. Primary infection occurs mainly in infants and young children and then the virus becomes latent in the dorsal root ganglion. It then is periodically reactivated throughout life causing mostly benign conditions.Microscopy, Electron, Transmission: Electron microscopy in which the ELECTRONS or their reaction products that pass down through the specimen are imaged below the plane of the specimen.Cytomegalovirus: A genus of the family HERPESVIRIDAE, subfamily BETAHERPESVIRINAE, infecting the salivary glands, liver, spleen, lungs, eyes, and other organs, in which they produce characteristically enlarged cells with intranuclear inclusions. Infection with Cytomegalovirus is also seen as an opportunistic infection in AIDS.Mesenchymal Stem Cell Transplantation: Transfer of MESENCHYMAL STEM CELLS between individuals within the same species (TRANSPLANTATION, HOMOLOGOUS) or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS).Microscopy, Fluorescence: Microscopy of specimens stained with fluorescent dye (usually fluorescein isothiocyanate) or of naturally fluorescent materials, which emit light when exposed to ultraviolet or blue light. Immunofluorescence microscopy utilizes antibodies that are labeled with fluorescent dye.Drug Compounding: The preparation, mixing, and assembling of a drug. (From Remington, The Science and Practice of Pharmacy, 19th ed, p1814)DNA, Viral: Deoxyribonucleic acid that makes up the genetic material of viruses.Flow Cytometry: Technique using an instrument system for making, processing, and displaying one or more measurements on individual cells obtained from a cell suspension. Cells are usually stained with one or more fluorescent dyes specific to cell components of interest, e.g., DNA, and fluorescence of each cell is measured as it rapidly transverses the excitation beam (laser or mercury arc lamp). Fluorescence provides a quantitative measure of various biochemical and biophysical properties of the cell, as well as a basis for cell sorting. Other measurable optical parameters include light absorption and light scattering, the latter being applicable to the measurement of cell size, shape, density, granularity, and stain uptake.Lipids: A generic term for fats and lipoids, the alcohol-ether-soluble constituents of protoplasm, which are insoluble in water. They comprise the fats, fatty oils, essential oils, waxes, phospholipids, glycolipids, sulfolipids, aminolipids, chromolipids (lipochromes), and fatty acids. (Grant & Hackh's Chemical Dictionary, 5th ed)Drug Stability: The chemical and physical integrity of a pharmaceutical product.Polyesters: Polymers of organic acids and alcohols, with ester linkages--usually polyethylene terephthalate; can be cured into hard plastic, films or tapes, or fibers which can be woven into fabrics, meshes or velours.Glioma: Benign and malignant central nervous system neoplasms derived from glial cells (i.e., astrocytes, oligodendrocytes, and ependymocytes). Astrocytes may give rise to astrocytomas (ASTROCYTOMA) or glioblastoma multiforme (see GLIOBLASTOMA). Oligodendrocytes give rise to oligodendrogliomas (OLIGODENDROGLIOMA) and ependymocytes may undergo transformation to become EPENDYMOMA; CHOROID PLEXUS NEOPLASMS; or colloid cysts of the third ventricle. (From Escourolle et al., Manual of Basic Neuropathology, 2nd ed, p21)Hemophilia B: A deficiency of blood coagulation factor IX inherited as an X-linked disorder. (Also known as Christmas Disease, after the first patient studied in detail, not the holy day.) Historical and clinical features resemble those in classic hemophilia (HEMOPHILIA A), but patients present with fewer symptoms. Severity of bleeding is usually similar in members of a single family. Many patients are asymptomatic until the hemostatic system is stressed by surgery or trauma. Treatment is similar to that for hemophilia A. (From Cecil Textbook of Medicine, 19th ed, p1008)Neoplasm Transplantation: Experimental transplantation of neoplasms in laboratory animals for research purposes.Flucytosine: A fluorinated cytosine analog that is used as an antifungal agent.Nucleoside Deaminases: Catalyze the hydrolysis of nucleosides with the elimination of ammonia.Brain Neoplasms: Neoplasms of the intracranial components of the central nervous system, including the cerebral hemispheres, basal ganglia, hypothalamus, thalamus, brain stem, and cerebellum. Brain neoplasms are subdivided into primary (originating from brain tissue) and secondary (i.e., metastatic) forms. Primary neoplasms are subdivided into benign and malignant forms. In general, brain tumors may also be classified by age of onset, histologic type, or presenting location in the brain.Peptides: Members of the class of compounds composed of AMINO ACIDS joined together by peptide bonds between adjacent amino acids into linear, branched or cyclical structures. OLIGOPEPTIDES are composed of approximately 2-12 amino acids. Polypeptides are composed of approximately 13 or more amino acids. PROTEINS are linear polypeptides that are normally synthesized on RIBOSOMES.RNA, Messenger: RNA sequences that serve as templates for protein synthesis. Bacterial mRNAs are generally primary transcripts in that they do not require post-transcriptional processing. Eukaryotic mRNA is synthesized in the nucleus and must be exported to the cytoplasm for translation. Most eukaryotic mRNAs have a sequence of polyadenylic acid at the 3' end, referred to as the poly(A) tail. The function of this tail is not known for certain, but it may play a role in the export of mature mRNA from the nucleus as well as in helping stabilize some mRNA molecules by retarding their degradation in the cytoplasm.Emulsions: Colloids formed by the combination of two immiscible liquids such as oil and water. Lipid-in-water emulsions are usually liquid, like milk or lotion. Water-in-lipid emulsions tend to be creams. The formation of emulsions may be aided by amphiphatic molecules that surround one component of the system to form MICELLES.Severe Combined Immunodeficiency: Group of rare congenital disorders characterized by impairment of both humoral and cell-mediated immunity, leukopenia, and low or absent antibody levels. It is inherited as an X-linked or autosomal recessive defect. Mutations occurring in many different genes cause human Severe Combined Immunodeficiency (SCID).Tissue Engineering: Generating tissue in vitro for clinical applications, such as replacing wounded tissues or impaired organs. The use of TISSUE SCAFFOLDING enables the generation of complex multi-layered tissues and tissue structures.Injections: Introduction of substances into the body using a needle and syringe.Tissue Distribution: Accumulation of a drug or chemical substance in various organs (including those not relevant to its pharmacologic or therapeutic action). This distribution depends on the blood flow or perfusion rate of the organ, the ability of the drug to penetrate organ membranes, tissue specificity, protein binding. The distribution is usually expressed as tissue to plasma ratios.Gliosarcoma: Rare mixed tumors of the brain and rarely the spinal cord which contain malignant neuroectodermal (glial) and mesenchymal components, including spindle-shaped fibrosarcoma cells. These tumors are highly aggressive and present primarily in adults as rapidly expanding mass lesions. They may arise in tissue that has been previously irradiated. (From Br J Neurosurg 1995 Apr;9(2):171-8)Injections, Intralesional: Injections introduced directly into localized lesions.Mice, SCID: Mice homozygous for the mutant autosomal recessive gene "scid" which is located on the centromeric end of chromosome 16. These mice lack mature, functional lymphocytes and are thus highly susceptible to lethal opportunistic infections if not chronically treated with antibiotics. The lack of B- and T-cell immunity resembles severe combined immunodeficiency (SCID) syndrome in human infants. SCID mice are useful as animal models since they are receptive to implantation of a human immune system producing SCID-human (SCID-hu) hematochimeric mice.Immediate-Early Proteins: Proteins that are coded by immediate-early genes, in the absence of de novo protein synthesis. The term was originally used exclusively for viral regulatory proteins that were synthesized just after viral integration into the host cell. It is also used to describe cellular proteins which are synthesized immediately after the resting cell is stimulated by extracellular signals.Receptors, Virus: Specific molecular components of the cell capable of recognizing and interacting with a virus, and which, after binding it, are capable of generating some signal that initiates the chain of events leading to the biological response.Genome, Viral: The complete genetic complement contained in a DNA or RNA molecule in a virus.Apoptosis: One of the mechanisms by which CELL DEATH occurs (compare with NECROSIS and AUTOPHAGOCYTOSIS). Apoptosis is the mechanism responsible for the physiological deletion of cells and appears to be intrinsically programmed. It is characterized by distinctive morphologic changes in the nucleus and cytoplasm, chromatin cleavage at regularly spaced sites, and the endonucleolytic cleavage of genomic DNA; (DNA FRAGMENTATION); at internucleosomal sites. This mode of cell death serves as a balance to mitosis in regulating the size of animal tissues and in mediating pathologic processes associated with tumor growth.Virus Integration: Insertion of viral DNA into host-cell DNA. This includes integration of phage DNA into bacterial DNA; (LYSOGENY); to form a PROPHAGE or integration of retroviral DNA into cellular DNA to form a PROVIRUS.Adenovirus E1 Proteins: The very first viral gene products synthesized after cells are infected with adenovirus. The E1 region of the genome has been divided into two major transcriptional units, E1A and E1B, each expressing proteins of the same name (ADENOVIRUS E1A PROTEINS and ADENOVIRUS E1B PROTEINS).Surface Properties: Characteristics or attributes of the outer boundaries of objects, including molecules.Liver: A large lobed glandular organ in the abdomen of vertebrates that is responsible for detoxification, metabolism, synthesis and storage of various substances.Tissue Scaffolds: Cell growth support structures composed of BIOCOMPATIBLE MATERIALS. They are specially designed solid support matrices for cell attachment in TISSUE ENGINEERING and GUIDED TISSUE REGENERATION uses.Lactic Acid: A normal intermediate in the fermentation (oxidation, metabolism) of sugar. The concentrated form is used internally to prevent gastrointestinal fermentation. (From Stedman, 26th ed)Injections, Intramuscular: Forceful administration into a muscle of liquid medication, nutrient, or other fluid through a hollow needle piercing the muscle and any tissue covering it.Transcription, Genetic: The biosynthesis of RNA carried out on a template of DNA. The biosynthesis of DNA from an RNA template is called REVERSE TRANSCRIPTION.Factor IX: Storage-stable blood coagulation factor acting in the intrinsic pathway. Its activated form, IXa, forms a complex with factor VIII and calcium on platelet factor 3 to activate factor X to Xa. Deficiency of factor IX results in HEMOPHILIA B (Christmas Disease).Micelles: Particles consisting of aggregates of molecules held loosely together by secondary bonds. The surface of micelles are usually comprised of amphiphatic compounds that are oriented in a way that minimizes the energy of interaction between the micelle and its environment. Liquids that contain large numbers of suspended micelles are referred to as EMULSIONS.Rabbits: The species Oryctolagus cuniculus, in the family Leporidae, order LAGOMORPHA. Rabbits are born in burrows, furless, and with eyes and ears closed. In contrast with HARES, rabbits have 22 chromosome pairs.Gene Silencing: Interruption or suppression of the expression of a gene at transcriptional or translational levels.Hematopoietic Stem Cells: Progenitor cells from which all blood cells derive.Endocytosis: Cellular uptake of extracellular materials within membrane-limited vacuoles or microvesicles. ENDOSOMES play a central role in endocytosis.Cercopithecus aethiops: A species of CERCOPITHECUS containing three subspecies: C. tantalus, C. pygerythrus, and C. sabeus. They are found in the forests and savannah of Africa. The African green monkey (C. pygerythrus) is the natural host of SIMIAN IMMUNODEFICIENCY VIRUS and is used in AIDS research.Blotting, Western: Identification of proteins or peptides that have been electrophoretically separated by blot transferring from the electrophoresis gel to strips of nitrocellulose paper, followed by labeling with antibody probes.Capsid Proteins: Proteins that form the CAPSID of VIRUSES.Gene Expression Regulation: Any of the processes by which nuclear, cytoplasmic, or intercellular factors influence the differential control (induction or repression) of gene action at the level of transcription or translation.Mutation: Any detectable and heritable change in the genetic material that causes a change in the GENOTYPE and which is transmitted to daughter cells and to succeeding generations.Pregnancy: The status during which female mammals carry their developing young (EMBRYOS or FETUSES) in utero before birth, beginning from FERTILIZATION to BIRTH.Reverse Transcriptase Polymerase Chain Reaction: A variation of the PCR technique in which cDNA is made from RNA via reverse transcription. The resultant cDNA is then amplified using standard PCR protocols.DNA Primers: Short sequences (generally about 10 base pairs) of DNA that are complementary to sequences of messenger RNA and allow reverse transcriptases to start copying the adjacent sequences of mRNA. Primers are used extensively in genetic and molecular biology techniques.Metal Nanoparticles: Nanoparticles produced from metals whose uses include biosensors, optics, and catalysts. In biomedical applications the particles frequently involve the noble metals, especially gold and silver.cis-trans-Isomerases: Enzymes that catalyze the rearrangement of geometry about double bonds. EC 5.2.Helper Viruses: Viruses which enable defective viruses to replicate or to form a protein coat by complementing the missing gene function of the defective (satellite) virus. Helper and satellite may be of the same or different genus.RNA, Viral: Ribonucleic acid that makes up the genetic material of viruses.Vaccines: Suspensions of killed or attenuated microorganisms (bacteria, viruses, fungi, protozoa), antigenic proteins, synthetic constructs, or other bio-molecular derivatives, administered for the prevention, amelioration, or treatment of infectious and other diseases.Adenoviridae Infections: Virus diseases caused by the ADENOVIRIDAE.Dogs: The domestic dog, Canis familiaris, comprising about 400 breeds, of the carnivore family CANIDAE. They are worldwide in distribution and live in association with people. (Walker's Mammals of the World, 5th ed, p1065)Materials Testing: The testing of materials and devices, especially those used for PROSTHESES AND IMPLANTS; SUTURES; TISSUE ADHESIVES; etc., for hardness, strength, durability, safety, efficacy, and biocompatibility.HEK293 Cells: A cell line generated from human embryonic kidney cells that were transformed with human adenovirus type 5.Chromosomes, Artificial, Human: DNA constructs that are composed of, at least, all elements, such as a REPLICATION ORIGIN; TELOMERE; and CENTROMERE, required for successful replication, propagation to and maintainance in progeny human cells. In addition, they are constructed to carry other sequences for analysis or gene transfer.Models, Animal: Non-human animals, selected because of specific characteristics, for use in experimental research, teaching, or testing.Immunohistochemistry: Histochemical localization of immunoreactive substances using labeled antibodies as reagents.Porosity: Condition of having pores or open spaces. This often refers to bones, bone implants, or bone cements, but can refer to the porous state of any solid substance.Cell Proliferation: All of the processes involved in increasing CELL NUMBER including CELL DIVISION.Phosphatidylethanolamines: Derivatives of phosphatidic acids in which the phosphoric acid is bound in ester linkage to an ethanolamine moiety. Complete hydrolysis yields 1 mole of glycerol, phosphoric acid and ethanolamine and 2 moles of fatty acids.Injections, Intravenous: Injections made into a vein for therapeutic or experimental purposes.Dextrans: A group of glucose polymers made by certain bacteria. Dextrans are used therapeutically as plasma volume expanders and anticoagulants. They are also commonly used in biological experimentation and in industry for a wide variety of purposes.Defective Viruses: Viruses which lack a complete genome so that they cannot completely replicate or cannot form a protein coat. Some are host-dependent defectives, meaning they can replicate only in cell systems which provide the particular genetic function which they lack. Others, called SATELLITE VIRUSES, are able to replicate only when their genetic defect is complemented by a helper virus.Mice, Transgenic: Laboratory mice that have been produced from a genetically manipulated EGG or EMBRYO, MAMMALIAN.Antineoplastic Agents: Substances that inhibit or prevent the proliferation of NEOPLASMS.Virus Latency: The ability of a pathogenic virus to lie dormant within a cell (latent infection). In eukaryotes, subsequent activation and viral replication is thought to be caused by extracellular stimulation of cellular transcription factors. Latency in bacteriophage is maintained by the expression of virally encoded repressors.Capsules: Hard or soft soluble containers used for the oral administration of medicine.Recombination, Genetic: Production of new arrangements of DNA by various mechanisms such as assortment and segregation, CROSSING OVER; GENE CONVERSION; GENETIC TRANSFORMATION; GENETIC CONJUGATION; GENETIC TRANSDUCTION; or mixed infection of viruses.Oligopeptides: Peptides composed of between two and twelve amino acids.Gold: A yellow metallic element with the atomic symbol Au, atomic number 79, and atomic weight 197. It is used in jewelry, goldplating of other metals, as currency, and in dental restoration. Many of its clinical applications, such as ANTIRHEUMATIC AGENTS, are in the form of its salts.Luciferases, Firefly: Luciferases from FIREFLIES, usually Photinus, that oxidizes FIREFLY LUCIFERIN to cause emission of PHOTONS.Tropism: The directional growth of an organism in response to an external stimulus such as light, touch, or gravity. Growth towards the stimulus is a positive tropism; growth away from the stimulus is a negative tropism. (From Concise Dictionary of Biology, 1990)Microscopy, Electron, Scanning: Microscopy in which the object is examined directly by an electron beam scanning the specimen point-by-point. The image is constructed by detecting the products of specimen interactions that are projected above the plane of the sample, such as backscattered electrons. Although SCANNING TRANSMISSION ELECTRON MICROSCOPY also scans the specimen point by point with the electron beam, the image is constructed by detecting the electrons, or their interaction products that are transmitted through the sample plane, so that is a form of TRANSMISSION ELECTRON MICROSCOPY.Botulism: A disease caused by potent protein NEUROTOXINS produced by CLOSTRIDIUM BOTULINUM which interfere with the presynaptic release of ACETYLCHOLINE at the NEUROMUSCULAR JUNCTION. Clinical features include abdominal pain, vomiting, acute PARALYSIS (including respiratory paralysis), blurred vision, and DIPLOPIA. Botulism may be classified into several subtypes (e.g., food-borne, infant, wound, and others). (From Adams et al., Principles of Neurology, 6th ed, p1208)Avidin: A specific protein in egg albumin that interacts with BIOTIN to render it unavailable to mammals, thereby producing biotin deficiency.Botulinum Toxins, Type A: A serotype of botulinum toxins that has specificity for cleavage of SYNAPTOSOMAL-ASSOCIATED PROTEIN 25.Cricetinae: A subfamily in the family MURIDAE, comprising the hamsters. Four of the more common genera are Cricetus, CRICETULUS; MESOCRICETUS; and PHODOPUS.Stem Cells: Relatively undifferentiated cells that retain the ability to divide and proliferate throughout postnatal life to provide progenitor cells that can differentiate into specialized cells.Fibroblasts: Connective tissue cells which secrete an extracellular matrix rich in collagen and other macromolecules.Quaternary Ammonium Compounds: Derivatives of ammonium compounds, NH4+ Y-, in which all four of the hydrogens bonded to nitrogen have been replaced with hydrocarbyl groups. These are distinguished from IMINES which are RN=CR2.Cell Division: The fission of a CELL. It includes CYTOKINESIS, when the CYTOPLASM of a cell is divided, and CELL NUCLEUS DIVISION.Polymerase Chain Reaction: In vitro method for producing large amounts of specific DNA or RNA fragments of defined length and sequence from small amounts of short oligonucleotide flanking sequences (primers). The essential steps include thermal denaturation of the double-stranded target molecules, annealing of the primers to their complementary sequences, and extension of the annealed primers by enzymatic synthesis with DNA polymerase. The reaction is efficient, specific, and extremely sensitive. Uses for the reaction include disease diagnosis, detection of difficult-to-isolate pathogens, mutation analysis, genetic testing, DNA sequencing, and analyzing evolutionary relationships.Targeted Gene Repair: A technique which uses synthetic oligonucleotides to direct the cell's inherent DNA repair system to correct a mutation at a specific site in an episome or chromosome.Capsid: The outer protein protective shell of a virus, which protects the viral nucleic acid.Combined Modality Therapy: The treatment of a disease or condition by several different means simultaneously or sequentially. Chemoimmunotherapy, RADIOIMMUNOTHERAPY, chemoradiotherapy, cryochemotherapy, and SALVAGE THERAPY are seen most frequently, but their combinations with each other and surgery are also used.Neoplasms, Experimental: Experimentally induced new abnormal growth of TISSUES in animals to provide models for studying human neoplasms.Transplantation, Heterologous: Transplantation between animals of different species.Cell Transplantation: Transference of cells within an individual, between individuals of the same species, or between individuals of different species.Injections, Intraocular: The administration of substances into the eye with a hypodermic syringe.DNA, Recombinant: Biologically active DNA which has been formed by the in vitro joining of segments of DNA from different sources. It includes the recombination joint or edge of a heteroduplex region where two recombining DNA molecules are connected.Fetal Therapies: Prenatal interventions to correct fetal anomalies or treat FETAL DISEASES in utero. Fetal therapies include several major areas, such as open surgery; FETOSCOPY; pharmacological therapy; INTRAUTERINE TRANSFUSION; STEM CELL TRANSPLANTATION; and GENETIC THERAPY.HIV-1: The type species of LENTIVIRUS and the etiologic agent of AIDS. It is characterized by its cytopathic effect and affinity for the T4-lymphocyte.Accidents, Traffic: Accidents on streets, roads, and highways involving drivers, passengers, pedestrians, or vehicles. Traffic accidents refer to AUTOMOBILES (passenger cars, buses, and trucks), BICYCLING, and MOTORCYCLES but not OFF-ROAD MOTOR VEHICLES; RAILROADS nor snowmobiles.Gels: Colloids with a solid continuous phase and liquid as the dispersed phase; gels may be unstable when, due to temperature or other cause, the solid phase liquefies; the resulting colloid is called a sol.Delivery of Health Care: The concept concerned with all aspects of providing and distributing health services to a patient population.Hemophilia A: The classic hemophilia resulting from a deficiency of factor VIII. It is an inherited disorder of blood coagulation characterized by a permanent tendency to hemorrhage.Cesarean Section: Extraction of the FETUS by means of abdominal HYSTEROTOMY.Solubility: The ability of a substance to be dissolved, i.e. to form a solution with another substance. (From McGraw-Hill Dictionary of Scientific and Technical Terms, 6th ed)Amino Acid Sequence: The order of amino acids as they occur in a polypeptide chain. This is referred to as the primary structure of proteins. It is of fundamental importance in determining PROTEIN CONFORMATION.PolyaminesNeovascularization, Physiologic: The development of new BLOOD VESSELS during the restoration of BLOOD CIRCULATION during the healing process.Viral Tropism: The specificity of a virus for infecting a particular type of cell or tissue.Oncolytic Virotherapy: Use of attenuated VIRUSES as ANTINEOPLASTIC AGENTS to selectively kill CANCER cells.Clinical Trials as Topic: Works about pre-planned studies of the safety, efficacy, or optimum dosage schedule (if appropriate) of one or more diagnostic, therapeutic, or prophylactic drugs, devices, or techniques selected according to predetermined criteria of eligibility and observed for predefined evidence of favorable and unfavorable effects. This concept includes clinical trials conducted both in the U.S. and in other countries.Antiviral Agents: Agents used in the prophylaxis or therapy of VIRUS DISEASES. Some of the ways they may act include preventing viral replication by inhibiting viral DNA polymerase; binding to specific cell-surface receptors and inhibiting viral penetration or uncoating; inhibiting viral protein synthesis; or blocking late stages of virus assembly.DNA-Binding Proteins: Proteins which bind to DNA. The family includes proteins which bind to both double- and single-stranded DNA and also includes specific DNA binding proteins in serum which can be used as markers for malignant diseases.
... (AAV) is a small virus which infects humans and some other primate species. AAV is not currently known to cause disease. The virus causes a very mild immune response, lending further support to its apparent lack of pathogenicity. Gene therapy vectors using AAV can infect both dividing and quiescent cells and persist in an extrachromosomal state without integrating into the genome of the host cell, although in the native virus some integration of virally carried genes into the host genome does occur. These features make AAV a very attractive candidate for creating viral vectors for gene therapy, and for the creation of isogenic human disease models. Recent human clinical ...
... is a virologist working as a professor at the Los Angeles City of Hope National Medical Center in the research department. Some of the viral areas she researches are: stem cells, gene therapy, genome editing, and parvovirus. Her main and current area of research is using Adeno-Associated Virus Vectors (AAV-Vectors). Additionally, she has had a role in many publications (see publications). Chatterjee received her B.Sc. in Cell & Molecular Biology from McGill University in Montreal, Canada. She continued on at McGill and received her Ph.D. in Immunology. Adeno-Associated Virus vectors can be used for stem cell gene therapy. Adenovirus' have a Baltimore classification of level I, meaning that they have a liner dsDNA genome within an icosahedral nucleocapsid. Some of their ...
... is the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency), a rare disorder caused by the absence of an essential protein called adenosine deaminase (ADA), which is required for the production of lymphocytes. Children born with ADA-SCID do not develop a healthy immune system so cannot fight off everyday infections, which results in severe and life-threatening illness. Without prompt treatment, the disorder often proves fatal within the child's first year of life. ADA-SCID is estimated to occur in approximately 15 patients per year in Europe. The treatment was developed at San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and developed by GlaxoSmithKline (GSK) ...
The year 2008 involved numerous significant scientific events and discoveries, some of which are listed below. 2 January - Researchers report that just four months of hormonal therapy before and with standard external beam radiation therapy can slow cancer growth by as much as eight years - especially the development of bone metastases - and increase survival rates in older men with potentially aggressive prostate cancer. ( 3 January - Gene therapy can reduce long-term drinking among rodents "An 'experiment of nature' is observed in some individuals of East Asian origin, who are 66 to 99 percent protected against alcoholism," explained Yedy Israel, professor of pharmacological and toxicological chemistry. ( 4 January - The National Science Foundation (NSF) and the National Aeronautics and Space Administration (NASA) jointly ...
... refers to the practice of altering the expression of a gene at one of various stages, with a view to alleviate some form of ailment. It differs from gene therapy in that gene modulation seeks to alter the expression of an endogenous gene (perhaps through the introduction of a gene encoding a novel modulatory protein) whereas gene therapy concerns the introduction of a gene whose product aids the recipient directly. Modulation of ...
Baculovirus gene transfer into Mammalian cells, known from scientific research articles as BacMam, is the use of baculovirus to deliver genes to mammalian cells. Baculoviruses are insect cell viruses that can be modified to express proteins in mammalian cells. The unmodified baculovirus is able to enter mammalian cells, however its genes are not expressed unless a mammalian recognizable promoter is incorporated upstream of a gene of interest. Both unmodified baculovirus and baculovirus modified with a mammalian promoter (BacMam) are unable to replicate in humans and are thus non infectious. Invented by Dr. Frederick M. Boyce, BacMam is a baculovirus-mediated gene transfer ...
Gene therapy utilizes the delivery of DNA into cells, which can be accomplished by several methods, summarized below. The two major classes of methods are those that use recombinant viruses (sometimes called biological nanoparticles or viral vectors) and those that use naked DNA or DNA complexes (non-viral methods). All viruses bind to their hosts and introduce their genetic material into the host cell as part of their replication cycle. This genetic material contains basic 'instructions' of how to produce more copies of these viruses, hacking the body's normal production machinery to serve the needs of the virus. The host cell will carry out these instructions and produce additional copies ...
A multiple cloning site (MCS), also called a polylinker, is a short segment of DNA which contains many (up to ~20) restriction sites - a standard feature of engineered plasmids. Restriction sites within an MCS are typically unique, occurring only once within a given plasmid. MCSs are commonly used during procedures involving molecular cloning or subcloning. Extremely useful in biotechnology, bioengineering, and molecular genetics, MCSs let a molecular biologist insert a piece of DNA or several pieces of DNA into the region of the MCS. This can be used to create transgenic organisms, also known as genetically modified organisms (GMOs). One bacterial plasmid used in genetic engineering as a plasmid cloning vector is pUC18. Its polylinker region is composed of several restriction enzyme recognition sites, that ...
In molecular biology, subcloning is a technique used to move a particular DNA sequence from a parent vector to a destination vector. Subcloning is not to be confused with molecular cloning, a related technique. Restriction enzymes are used to excise the gene of interest (the insert) from the parent. The insert is purified in order to isolate it from other DNA molecules. A common purification method is gel isolation. The number of copies of the gene is then amplified using polymerase chain reaction (PCR). Simultaneously, the same restriction enzymes are used to digest (cut) the destination. The idea behind using the same restriction enzymes is to create complementary sticky ends, which will facilitate ligation later on. A phosphatase, commonly calf-intestinal alkaline phosphatase (CIAP), is also added to prevent self-ligation of the destination vector. The digested ...
Dual expression recombinase based (DERB) single vector system is a method of efficient cloning and subcloning of plasmid vectors for high throughput screening (HTS) and verification of protein-protein interactions inside living cells. DERB was developed by Lu JP et al. Plasmid Vectors are deliberately constructed circular double-strand DNA loops capable of self-amplification and protein production. They are widely used in laboratories and the bio-medical and pharmaceutical industries to produce of DNA or protein in quantity. The DERB vector system consists of a series of vectors, each of which produces two or more proteins which are labeled or tagged for screening and verification of molecular interactions such as protein-protein interactions. The following characteristics of the vectors ensure highly efficient cloning or subcloning of the protein of interest ORFs into the DERB vectors for high-throughput screening (HTS) and verification of protein-protein interactions after single introducing ...
A short hairpin RNA or small hairpin RNA (shRNA/Hairpin Vector) is an artificial RNA molecule with a tight hairpin turn that can be used to silence target gene expression via RNA interference (RNAi). Expression of shRNA in cells is typically accomplished by delivery of plasmids or through viral or bacterial vectors. shRNA is an advantageous mediator of RNAi in that it has a relatively low rate of degradation and turnover. However, it requires use of an expression vector, which can pose safety concerns. The promoter choice is essential to achieve robust shRNA expression. At first, polymerase III promoters such as U6 and H1 were used; however, these promoters lack spatial and temporal control. As such, there has been a shift to using polymerase II promoters to regulate shRNA expression. Expression of shRNA in cells can be obtained by delivery of plasmids or through ...
An expression cassette is a distinct component of vector DNA consisting of a gene and regulatory sequence to be expressed by a transfected cell. In each successful transformation, the expression cassette directs the cell's machinery to make RNA and protein(s). Some expression cassettes are designed for modular cloning of protein-encoding sequences so that the same cassette can easily be altered to make different proteins. An expression cassette is composed of one or more genes and the sequences controlling their expression. An expression cassette comprises three components: a promoter sequence, an open reading frame, and a 3' untranslated region that, in eukaryotes, usually contains a polyadenylation site. Different expression cassettes can be transfected into different organisms including bacteria, yeast, plants, and mammalian cells as long as the correct regulatory ...
The reset vector is the default location a central processing unit will go to find the first instruction it will execute after a reset. The reset vector is a pointer or address, where the CPU should always begin as soon as it is able to execute instructions. The address is in a section of non-volatile memory initialized to contain instructions to start the operation of the CPU, as the first step in the process of booting the system containing the CPU. The reset vector for the 8086 processor is at physical address FFFF0h (16 bytes below 1 MB). The value of the CS register at reset is FFFFh and the value of the IP register at reset is 0000h to form the segmented address FFFFh:0000h, which maps to physical address FFFF0h. The reset vector for the 80286 processor is at physical address 00FFFF0h (16 bytes below 1 MB). The value of the CS register at reset is F000h and the value of the IP register at reset is FFF0h to form the segmented address F000h:FFF0h, which maps to physical address 00FFFF0h in ...
Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has ... This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows AMT ... AMT is a world leader in the development of human gene based therapies. AMT has a product pipeline of gene therapy products in ... a leader in the field of human gene therapy, announced today data demonstrating that one-time administration of the gene ...
... of gene therapy is to use a modified virus-a viral vector-as a vehicle to deliver healthy genes into a cell to replace faulty ... Voyager was founded based around advances in developing adeno-associated viruses, or AAVs, as delivery tools for gene therapy ... that are supposed to either replace a faulty gene, or shut down a disease-causing one. ... are using to deliver gene therapies.. Third Rock wanted to start a gene therapy company that could target central nervous ...
Using adeno-associated viral vectors as the delivery vehicle of choice for therapeutic genes, the company has designed and ... diseases that are caused by one faulty gene. AMT currently has a product pipeline with nine products at different stages of ... AMT has a unique production platform that circumvents the obstacles that have hindered development of gene therapy technologies ... The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients ...
Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able ... diseases that are caused by one faulty gene. AMT currently has a product pipeline with six products at different stages of ... gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy to ... a leader in the field of human gene therapy, today announced that it will release its financial results for the first six ...
Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able ... diseases that are caused by one faulty gene. AMT currently has a product pipeline with six products at different stages of ... Exclusive license to all gene therapy products from CIMA, one of Europes leading gene therapy centers ... gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy to ...
... a leader in the field of human gene therapy, announced today... ... vectors as the delivery vehicle of choice for therapeutic genes ... This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows AMT ... AMTs hemophilia B program, which consists of an adeno-associated viral (AAV) vector containing the human factor IX gene, is ... AMT is a world leader in the development of human gene based therapies. AMT has a product pipeline of gene therapy products in ...
PRNewswire/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today reported ... Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has ... This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows AMT ... AMT is a world leader in the development of human gene based therapies. The companys lead product Glybera(R), a gene therapy ...
... a leader in the development of gene based therapies, today announced that its gene… ... diseases that are caused by one faulty gene. Currently, AMT has a product pipeline with several AAV-based gene therapy products ... Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has ... a leader in the development of gene based therapies, today announced that its gene therapy product incorporating siRNA ...
... a leader in the field of human gene therapy, announced today that Dr. Ronald H.W.… ... Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able ... diseases that are caused by one faulty gene. AMT currently has a product pipeline with nine products at different stages of ... gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from ...
Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has ... This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene, and allows ... uniQure is a world leader in the development of human gene based therapies. uniQures Glybera, a gene therapy for the treatment ... and is the first approved gene therapy in the Western world. uniQures product pipeline of gene therapy products in development ...
To date genetically altered viruses are commonly used as gene delivery vehicle (vector) which has an advantage of evolutionary ... transfer of therapeutic or working copy of genes into a specific cell of an individual in order to repair a faulty copy of gene ... Non viral vectors which include the physical transfection of genes can be accomplished by electrophoration, microinjection, or ... Oral cancer remains one of the leading causes of death world wide. Various means to destroy tumor cells preferentially have ...
... researchers at Thomas Jefferson University describe how they can transfer genes into brain neurons intravenously, usi ... using a viral gene delivery vehicle (vector) that causes no side effects. ... or potentially correct selected disorders that are due to one faulty gene," Dr. Strayer says. "Our approach also allows us to ... Using mannitol before delivery of the gene-laden SV40 viral vectors in mice also directs most of the viral particles into the ...
Stanford University School of Medicine researchers have demonstrated that gene therapy can be effective without causing a ... dangerous side effect common to all gene therapy: an autoimmune reaction to the normal protein, which the patients immune ... The abridged gene fits snugly into a viral delivery vehicle designed some time ago by Jeffrey Chamberlain, PhD, a co-author of ... have demonstrated that gene therapy can be effective without causing a dangerous side effect common to all gene therapy: an ...
MIT researchers have developed nanoparticles that can deliver the CRISPR genome-editing system and specifically modify genes in ... Using the new delivery technique, the researchers were able to cut out certain genes in about 80 percent of liver cells, the ... Using a new gene-editing system based on bacterial proteins, MIT researchers have cured mice of a rare liver disorder caused by ... Explore further: Curing disease by repairing faulty genes More information: Structure-guided chemical modification of guide RNA ...
Repairing the faulty genes that cause haemophilia could ultimately cure the disease, but it will be a tough challenge ... Repairing the faulty genes that cause haemophilia could ultimately cure the disease, but it will be a tough challenge ... So although gene therapy could be a one-off cure, if the immune response is triggered before the therapy reaches the target, it ... The viral vehicle AAV8 is ideal for treating haemophilia B, but it works less well for haemophilia A. This is because the DNA ...
In these cases, the patient's own bone marrow cells can be removed and the faulty gene corrected with gene therapy. The ... Drawbacks of viral vectors:. *They can carry a limited amount of genetic material. Therefore, some genes may be too big to fit ... Scientists refer to these DNA delivery vehicles as vectors.. There is no perfect vector that can treat every disorder. Like ... Gene Delivery: Tools of the Trade. Retrieved December 13, 2018, from ...
... like suicide gene therapy, offer potential improvements in the personalized treatment of mesothelioma cancer. ... Second, inserting these tumor-suppressing genes requires a microscopic delivery vehicle, or vector, that can penetrate deep ... Inserting Tumor-Suppressing Genes. The most obvious gene therapy approach is to fix the genetic fault that causes cells to ... Many researchers believe that just as faulty genes are the key to cancer formation, modified genes may be the key to cancer ...
VIRAL VECTORS. Viruses have many advantages as vehicles for the delivery of genes, as this is their normal function. There have ... Recently, a novel technique has been devised to correct a faulty gene by harnessing the repair mechanisms of the host. In this ... true for dominant negative mutations where simple introduction of a normal gene product may not overcome the dysfunction caused ... Methods of gene delivery to the liver. The importance to successful gene therapy of developing effective gene delivery systems ...
AAV does not contain any viral genes and contains only the therapeutic gene and it does not integrate into the genome. It ... Depending upon the method of correcting the faulty gene, gene therapy can be classified into the following categories:. *Gene ... Gene therapy is believed by many to be the therapy of the twenty first century because it aims to eradicate cause rather than ... Viral Vectors: Natures Own Infecting Vehicles. Viruses have evolved specific mechanisms through the course of evolution to ...
... we need delivery vehicles that will safely carry CRISPR cargos to the right addresses-vehicles such as viral vectors and ... Deploying viral vehicles. Adeno-associated virus (AAV) has established itself as a vehicle of choice for gene therapy, and many ... receiving direct injections of viral particles laden with genes encoding the CRISPR-Cas9 machinery to correct a retinal gene ... Using HDR] can cause acute toxicity inside cells, which dislike having a lot of donor DNA in there." ...
"CRISPR-Gold was able to correct disease-causing gene mutations in vivo, via the non-viral delivery of Cas9 protein, guide RNA ... "CRISPR-Gold is the first example of a delivery vehicle that can deliver all of the CRISPR components needed to correct gene ... precise and easy-to-use gene editor, researchers have hoped that therapies based on CRISPR-Cas9 would one day revolutionize the ... which is a significant improvement over previously published approaches that only removed the faulty part of the gene, making ...
The ability to silence specific genes, thereby ridding the body of pesky disease-causing proteins, also could provide ... is an attractive target since the organ has a natural propensity to take up the nanolipid delivery vehicles created by Alnylam ... The trial showed that the therapy was safe, and biopsies from some of the volunteers tumors showed the RNAi was doing its job ... The ability to silence any gene in the body would prove to be an incredible boon to research. No longer would scientists ...
... consequences of DYSTROPHIN deficiency in DMD aim to restore its biological function through viral-mediated delivery of genes ... In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector. Cell Reports 12, 1056-1068 ( ... For certain disorders such as muscular dystrophy, it may be possible to achieve therapeutic benefit by editing the faulty gene ... or duplications in the DMD gene that cause genetic frame-shift or loss of protein expression (1). Efforts under development to ...
Gene Overdose Causes Extreme Thinness. Scientists have discovered a genetic cause of extreme thinness for the first time. ... Poloxamers have been used before as a vehicle for delivering drugs, including chemotherapeutics, vaccines and anti-viral ... Gene Defect Linked to Disfiguring Disorder. The faulty gene responsible for Proteus syndrome, a rare disorder of uncontrolled ... Parents Stress Leaves Marks on Childrens Genes. Epigenetics changes the expression of genes, and can induce long lasting ...
... long-term expression of the missing gene products. They develop to become cardiomyocytes in the infracted myocardium. Myoblasts ... Non-viral approaches using lipofection, nanoparticles and plasmids (naked RNA) account for about 40% of gene therapy studies. ... in addition to just replenishing the missing gene(s) or normal copies of the aberrant gene(s). The replacement genes then ... Central to this technology is the use of viruses as vectors to deliver normal copies of the faulty or missing gene into a ...
  • The company would do so by injecting modified viruses into the spine or brain containing either specific proteins, or micro RNA silencing molecules, that are supposed to either replace a faulty gene, or shut down a disease-causing one. (
  • Using such a method, it may be possible, for example, to treat diseases thought to be due to excessive oxidative damage to neurons and neuron proteins, such as early Alzheimer's disease or Parkinson's disease, with extra genes that limit oxidative damage to brain neurons, he says. (
  • Synthetic vectors called virosomes are essentially liposomes covered with viral surface proteins. (
  • The viral proteins interact with proteins on the target-cell surface, helping the virosome fuse with the cell membrane and dump its contents into the cell. (
  • Different types of viral proteins can target specific types of cells. (
  • Genes are coded instructions for making proteins, the molecules that control how cells work. (
  • Every cell requires a host of genes that act as blueprints of all the proteins essential for its proper functioning. (
  • Since most diseases are the result of problematic proteins - either faulty construction of a necessary protein or too much of a good thing - ridding cells of certain proteins might lessen their consequences or, in the case of some infectious diseases, cure certain conditions altogether. (
  • The ability to silence specific genes, thereby ridding the body of pesky disease-causing proteins, also could provide unprecedented gains for therapies. (
  • The 2-calendar year flare-free success was 80% with biologic remedies in comparison to 43% in non-biologic therapies (transient ischemic strike, erythrocyte sedimentation price, C-reactive proteins, von Willebrand aspect antigen. (
  • The mRNA and DNA precursors of proteins, however, are promising therapeutically in that they can be specifically targeted via Watson-Crick base pairing and, in the case of gene editing, which aims to permanently change the host's DNA, represent an avenue to cure a genetic defect as opposed to just treating it. (
  • The T cells are extracted and genetically altered so that they have a new gene that codes for a protein, known as a chimeric antigen receptor (CAR), that is a hybrid of two immune system proteins. (
  • Mueller said that the best guess now is that there are 400-500 genes and proteins responsible for genetic deafness. (
  • The gene, or combination of genes formed by these base pairs ultimately direct an organism's growth and characteristics through the production of certain chemicals, primarily proteins, which carry out most of the body's chemical functions and biological reactions. (
  • Alternatively, PAD enzymes may leak out of the dying cells, become PF-04929113 activated (the extracellular calcium concentration is approximately 10-3 mol/l, which is sufficient for PAD activity ), and cause citrullination of extracellular proteins. (
  • Additionally, XPO5 can acknowledge and export organized RNAs that are unrelated to pre-miRNAs, which includes viral mini-helix RNA and tRNA, along with specific various other proteins, such as for example STAU2, ILF3, and JAZ [7, (
  • IL2RG, RAG1 and/or RAG2 gene) into the genome of a cell for provision of proteins lacking or deficient in SCID. (
  • Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today data demonstrating that one-time administration of the gene therapy Glybera® (alipogene tiparvovec) is able to markedly improve chylomicron (fat particles in the blood) metabolism following consumption of a low fat meal. (
  • BLUE ) is using in its clinical trials , or modified AAV vectors, which startups like Dimension Therapeutics , Audentes Therapeutics , and others, are using to deliver gene therapies. (
  • AMSTERDAM, The Netherlands, June 18 /PRNewswire/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today new data showing that a one-time administration of its lead product GlyberaTM results in significant long-term health benefits. (
  • AMSTERDAM, August 22 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it will release its financial results for the first six months of 2007 on Wednesday, August 29, 2007 at 8 am Central European Time (CET). (
  • AMSTERDAM, August 29 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today reported its results for the first half year of 2007. (
  • Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its gene therapy program for the treatment of hemophilia B. Orphan designation in the U.S. could provide up to seven years market exclusivity on regulatory approval. (
  • AMSTERDAM, February 16, 2011 /PRNewswire/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today reported its results for the year to December 31, 2010. (
  • AMSTERDAM - Amsterdam Molecular Therapeutics (AMT) Holding N.V. (Euronext: AMT), a leader in the development of gene based therapies, today announced that its gene therapy product incorporating siRNA sequences into microRNA scaffolds to silence Apolipoprotein B100 (AAV-miApoB) was able to significantly lower plasma cholesterol levels in vivo over a period of 18 weeks. (
  • Amsterdam, The Netherlands - Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that Dr. Ronald H.W. Lorijn will for personal reasons retire as chief executive officer, effective February 1, 2009. (
  • Fidelity Biosciences, a venture capital firm that is a subsidiary of Fidelity Investments, and REGENX Biosciences, announced the formation of Dimension Therapeutics, a Cambridge gene therapy company focused on developing treatments for rare diseases such as hemophilia. (
  • We are gearing up for a clinical trial with a company we licensed the therapy to, Orchard Therapeutics,' says Bigger. (
  • In the same year, sickle cell anaemia mice were treated by introducing the artificial therapeutics gene. (
  • With her co-founders, George Foot and Zuzanna Brzosko , they are developing Sixfold's unique Programmable Oligonucleotide Delivery Devices (PODDs), which aim to deliver gene therapy molecules exclusively to diseased cells. (
  • However, when these cells attack the LDL molecules, they cause plaque deposits on the walls of the artery. (
  • Recent trials show that therapies Isoprenaline HCl directed against these molecules can improve anti-cancer immunity. (
  • In HIV-1 infection T cell exhaustion is associated with the up-regulation of surface molecules called immune checkpoint receptors (ICRs) such as PD-1, Tim-3 and Lag-3 [12,17C which have also been associated with the size of the HIV reservoir and time to viral rebound after therapy cessation[21, (
  • Mutations in the human version of the gene are associated with a rare disorder called dominant familial hypercholesterolemia, and the FDA recently approved two antibody drugs that inhibit Pcsk9. (
  • This is particularly true for dominant negative mutations where simple introduction of a normal gene product may not overcome the dysfunction caused by the continued production of an abnormal product. (
  • In about a third of patients, the gene for dystrophin has small deletions or single base mutations that render it nonfunctional, which makes this gene an excellent candidate for gene editing. (
  • This can then be used to introduce either mutations, knock-ins, knock-outs, or replace a faulty piece of DNA with a correct one. (
  • The present disclosure is in the field of genome engineering, particularly targeted integration of a functional SCID-related genes (e.g. (
  • After several years of it, in 1971, Carl R. Merril experimented on Human Fibroblast cells and concluded that DNA could be inserted into the human genome for fixing the mutant gene. (
  • After several years of it, in 1971, Carl R. Merril experimented on Human Fibroblast cells and concluded that DNA could be inserted in the human genome for fixing the mutant gene. (
  • Successful HSC GT requires inserting the therapeutic gene into the cellular chromosomal DNA, or editing a selected nucleotide sequence, to ensure maintenance of gene correction as the cell replicate its genome and transmit it to the progeny, whether during self‐renewal or in the output of differentiating cell lineages. (
  • There were techniques to exploit the HIV-1 virus to engineer vectors for gene transfer, the combining of viruses with polymers or cationic lipids to improve gene transfer, the attachment of nuclear localization signal peptides to oligonucleotides to direct them to nuclei, and the invention of molecular switch systems allowing genes to be turned on or off at will . (
  • 3 This was achieved by segmental hepatic resection, preparation of hepatocyte cultures, and transduction of these cultures with a recombinant retrovirus encoding the gene for the human LDL receptor. (
  • Successful human gene therapy requires methods to transfer recombinant genes to cells efficiently. (
  • The second, called ex vivo (ex VEE-voh), is to deliver the gene to cells that have been removed from the body and are growing in culture. (
  • Several gene therapy successes use ex vivo gene delivery as an alternative to bone marrow transplants. (
  • 2 In clinical trials, five patients homozygous for familial hypercholesterolaemia underwent ex vivo replacement of the faulty gene. (
  • This study served to demonstrate the feasibility of ex vivo therapy, although concerns remain regarding the long term efficacy of this approach. (
  • The paradigm for in vivo therapy has been the approach to ornithine transcarbamylase (OTC) deficiency. (
  • Another recent in vivo experiment demonstrated the use of retroviral vectors to produce sustained expression of therapeutic levels of factor VIII in a neonatal mouse model of haemophilia A. 6 As it is desirable to institute gene therapy early in life, the propagation of neonatal hepatocytes represents a promising approach with clinical relevance. (
  • However, this method is clearly not suitable for manipulating large numbers of cells in living tissue, and most in vivo work is instead focused on the use of viral vectors or synthetic nanoparticles to achieve targeted delivery. (
  • I developed a patented method to generate antigenic diversity in vivo without killing the cells as a vaccine strategy to avoid viral escape. (
  • Using in vivo and in vitro imaging of neuronal processes in a mouse model lacking one Lis1 gene copy we demonstrate a prominent Lis1 role in the dynamic behaviour of protrusions important for synapse formation and maturation. (
  • A schematic representation of HSC GT showing the crucial steps of the process and its potential clinical applications: (1) HSPC are harvested from the mobilized peripheral blood or bone marrow of a patient and (2) cultured ex vivo in suitable conditions allowing maintenance or expansion of the rare cells with long‐term repopulating potential, while they are subjected to gene transfer or gene editing. (
  • The patient is then administered a conditioning regimen which depletes endogenous HSPC from the bone marrow and makes space for her/is ex vivo engineered cells, which are then infused back (autologous cell therapy). (
  • AMT has a product pipeline of gene therapy products in development for hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. (
  • Our collaboration with FIMA in Pamplona, Spain, to develop a gene therapy for acute intermittent porphyria (AIP) is progressing very well, and has generated positive pre-clinical data. (
  • The Cas9 enzyme then cuts the DNA at that precise location, allowing for genes to be turned on or off or for the removal or insertion of DNA. (
  • It is based on a viral defence mechanism found in certain bacteria, and uses an endonuclease (Cas9) guided by a single-RNA to precisely target and cut complementary genomic sequences. (
  • But Voyager will be using it to pursue a big effort: to use gene therapy, and to a certain extent, micro RNA tools, to try to either cure, or significantly reverse the effects of a wide range of serious central nervous system disorders like Lou Gehrig's Disease, Friedreich's Ataxia, and Parkinson's. (
  • These horrible CNS disorders that no one can do anything with, gene therapy really is going to be the answer, we believe, to a lot of these," says Mark Levin, the co-founder of Third Rock, and Voyager's interim CEO. (
  • They say their findings potentially represent a major advance in the effort to treat brain disorders with therapeutic transgenes, or external genes - suggesting that gene therapy to the brain could be given to patients on an outpatient basis, simply by IV administration into the arm. (
  • This is theoretical of course, but through our method of delivering genes directly to neurons, we might be able to arrest or slow the progression of a number of neurodegenerative disorders, or potentially correct selected disorders that are due to one faulty gene," Dr. Strayer says. (
  • The new nanoparticles permanently edit the gene following a single treatment, and the technique also offers promise for treating other liver disorders, according to the MIT team. (
  • What disorders have been or might be soon treated with gene therapy? (
  • Current hypotheses regarding neurological and neurodevelopmental disorders, such as schizophrenia, autism and various forms of mental retardation, include not only formation of faulty synaptic contacts, but also the failure to successfully prune synapses once formed and the inability to make new connections during childhood development. (
  • This drawback from the microorganisms that travel immunoregulatory circuits leads to faulty immunoregulation that, with regards to the hereditary history of any provided individual, can express as a number of chronic inflammatory disorders, including allergy symptoms, IBD and autoimmunity. (
  • So many other disorders are adding every day in the list of gene therapy and that is why a successful gene targeting technology, a gene therapy, is a must needed technology in forthcoming years for mankind. (
  • Experiment on the model organism demonstrated that the use of successful gene therapy for hereditary disorders are still a long way off. (
  • Various different monogenic and polygenic disorders are evolving every day that is why a successful gene targeting technology- a gene therapy, is a must needed technology in forthcoming years for mankind. (
  • I rectified a problem with a diagnostic procedure that was causing false positive results for a hospital associated bacterial infection Clostridium difficile. (
  • This study measures three exhaustion markersPD-1, Tim-3, Lag-3 Cin individuals with HIV Isoprenaline HCl recruited to a randomised controlled trial of therapy in early HIV infection called SPARTAC. (
  • Successful hepatocyte-specific delivery of microRNA (miRNA) and significant demonstration of gene silencing again illustrates the strength of AMT's AAV platform. (
  • The p53 gene , the BAP1 gene and microRNA gene 16 have all been studied as genes that may be able to stop the progression of mesothelioma. (
  • Provided strong evidence implicating an important role of altered microRNA expression in cancer initiation and progression, the genes responsible for microRNA biogenesis may also play a role in tumorigenesis. (
  • It wasn't until recently in 2017 that the U.S. Food and Drug Administration (FDA) approved a gene-therapy-based cancer treatment for the first time. (
  • The technology's potential to address unmet medical needs and to cure disease results from its demonstrated ability to permanently silence genes which cause the condition. (
  • There, the two scientists wrote about a strange phenomenon whereby double-stranded RNA injected into C. elegans had the power to potently silence genes. (
  • Scientists refer to these DNA delivery 'vehicles' as vectors . (
  • But scientists have actually been able to use viruses to deliver DNA to cells for gene therapy. (
  • No longer would scientists patiently have to wait a year or longer to make knockout mice to study gene function - with RNAi, the knockout could happen instantly. (
  • To overcome these challenges, the Berkeley scientists invented a delivery vessel that binds all of these components together, and then releases them when the vessel is inside a wide variety of cell types, triggering homology directed repair. (
  • Even scientists do not know how effective these therapies are becoming. (
  • The scientists tested their assumption in mice whose Dickkopf-1 gene is permanently silenced. (
  • Scientists have known how to manipulate a gene's structure in the laboratory since the early 1970s through a process called gene splicing. (
  • The idea of gene transfer in a human was given by Martine Cline, he was one of the pioneer scientists in gene therapy research. (
  • Your memory is either faulty or you did not see that I took Sinemet and Symmetrel for the first 2 years before being taken off those and put onto Eldepryl. (
  • U.S. orphan designation provides additional support for our hemophilia B gene therapy program and supplements the designation in the EU received in November,' said Jörn Aldag, CEO of AMT. (
  • These genes have been used successfully in Dr. Strayer's laboratory to treat some of the manifestations of HIV-related injury to neurons, which is a result of oxidative damage is important to loss of cells. (
  • In 2002, the scientist had successfully treated adenosine deaminase deficiency through gene therapy. (
  • beta thalassemia major child was successfully treated with gene therapy. (
  • In 1961, K Lorraine had successfully introduced a functional gene in the mammalian cells. (
  • Recent developments in nanotechnology have shown that nanocarriers, due to their small sizes, exhibit optimized physicochemical and biological properties that make them easily taken up by cells so that they can be successfully used as delivery tools for currently available therapeutic agents 7 . (
  • This strategy involves administration of a normal gene to replace a missing or dysfunctional gene product resulting from a defective gene, as has been illustrated by studies on the hereditary disorder familial hypercholesterolaemia. (
  • So although gene therapy could be a one-off cure, if the immune response is triggered before the therapy reaches the target, it is useless. (
  • The advantages are that they can carry larger genes, and most don't trigger an immune response. (
  • In general terms, a gene is a segment of nucleic acid that, taken as a whole, specifies a trait. (
  • This progress comes amidst a general surge in progress with nucleic acid-based therapies. (
  • As such, the nucleic acid delivery field has centered on the design of delivery methods and materials that will transport RNA drugs to the site of interest. (
  • In tests for spatial orientation and memory, mice in advanced adult age whose Dickkopf gene had been silenced reached an equal mental performance as young animals. (
  • Finally, Lis1 +/− and Lis1cko mutant mice showed deficits in social interactions, establishing a link between Lis1 gene function and autistic‐like behaviours in mice. (
  • The tumor suppressor gene is essential for embryonic development, as nullizygous mice die in midgestation (6, 22, 25). (
  • Dr Peter French , CEO of Benitec Biopharma, commented, "Benitec Biopharma is very pleased to have executed this licensing agreement with uniQure, the first company to achieve market approval for a gene therapy product, Glybera, in the West. (
  • uniQure's Glybera, a gene therapy for the treatment of lipoprotein lipase deficiency has been approved in the European Union, and is the first approved gene therapy in the Western world. (
  • In 2015, Tuddenham and his team hope to lead trials to test safety and efficacy of their optimized factor VIII gene therapy for people with haemophilia A. The number of people in the trials is likely to be between 10 and 20, but even if the factor is expressed effectively in humans, there are still hurdles to overcome. (
  • Although gene therapy testing in humans has advanced rapidly, many questions surround its use. (
  • LIS1 ( PAFAH1B1 ) mutation can impair neuronal migration, causing lissencephaly in humans. (
  • In humans, a 50% loss of LIS1 causes lissencephaly as a result of aberrant neuronal migration and organization. (
  • China was the pioneer in gene therapy, they had approved first gene therapy in humans. (
  • Little messengers called mRNA make copies of the gene blueprints, edits them and carry them to "builders" called ribosomes. (
  • mRNA appearance from the related genes was normalized to cyclophilin. (
  • A gene is the basic unit of heredity in a living organism . (
  • Genes control heredity and provide the basic biological code for determining a cell's specific functions. (
  • Gene therapy has grown out of the science of genetics or how heredity works. (
  • The gene encoding dystrophin is far too big for a gene-hauling virus to take onboard. (
  • Fortunately, a mere fraction of the entire gene is enough to generate a reasonably functional version of dystrophin, called microdystrophin. (
  • In this paper, we were actually able to correct [the gene for] dystrophin back to the wild-type sequence" via homology-directed repair (HDR), coauthor Niren Murthy, a drug delivery researcher at the University of California, Berkeley, tells The Scientist. (
  • The present disclosure provides methods and compositions for enhanced delivery of siRNA or miRNA, into the interior of multilayered tissues, and into the cytoplasm or nucleus of cells of a tissue. (
  • Such methods and compositions yield tumor-selective and intracellular delivery of RNAi agents and allow for RNAi-mediated activity such as knock-down of the target genes and associated products. (
  • In particular, the present invention provides benzodiazepine derivatives and methods of using benzodiazepine derivatives as therapeutic agents to treat a number of conditions associated with the faulty regulation of the processes of programmed cell death, autoimmunity, inflammation, and hyperproliferation, and the like. (
  • 7 . Quantitation of GAD67 Gene Expression in Prefrontal Cortex of Schizophrenia Patients Using the iCycler iQ Detection System and Molecular Beacons, Rev A 8 . (
  • They found that mannitol increased expression of the gene ten-fold in the brain and spinal cord. (
  • Since then, Tuddenham has not only been trying to fix the gene but also to improve its expression. (
  • In this disease, a defect in the low density lipoprotein (LDL) receptor gene results in abnormal expression of the LDL receptor and consequent failure of clearance of LDL cholesterol. (
  • Expression of the introduced gene relieves/eliminates symptoms of the disorder, but this effect is not heritable. (
  • Epigenetics changes the expression of genes, and can induce long lasting changes in our children when they are exposed our to stress. (
  • Reporter gene expression was detected up to 28 days post-injection. (
  • Since endosome rupture is a rate-limiting step in foreign gene expression, we developed a two-step assay to quantitative virus-mediated membrane rupture. (
  • The effect of PD-1 and Lag-3 expression on CD8 Isoprenaline HCl T cells retained statistical significance in Cox proportional hazards models including antiretroviral therapy and CD4 count, but not pVL as co-variants. (
  • 22. The non-human transgenic animal of claim 21, wherein the functional disruption substantially reduces the expression of the Rgs16 and Rgs8 gene products. (
  • 26. The non-human transgenic animal of claim 23, wherein said misexpression results in decreased expression of one or more tumor suppressor genes or loci. (
  • In concordat, a quantitative real-time PCR review of room cycle genes confirmed hyper-expression of Cdk1, a gene regulating the G1 to S and G2 to M transition of the stall run, and Nestin, a marker of neural prow cells and neural progeni- tor cells. (
  • Benitec Biopharma Ltd is developing novel treatments for chronic and life-threatening conditions based on targeted gene-silencing activity using a transformational technology: DNA-directed RNA interference (ddRNAi) - sometimes called expressed RNAi. (
  • Of the six patients who enrolled, four were able to discontinue their infusion treatments after the therapy. (
  • Treatments for Dry Macular Degeneration: An Unmet Need Age-Related Macular Degeneration (AMD) is the leading cause of legal blindness in Americans over 65. (
  • Because CARs can induce T cells to attack tumors in an MHC-unrestricted manner, the application of adoptive T-cell therapy in cancer treatments has expanded. (
  • In the traditional procedure, a chemotherapy drug kills bone marrow cells to make a space for stem cells from a donor without the faulty gene. (
  • Progress in bone marrow transplants over recent decades has greatly assisted such therapies. (
  • The gene‐modified cells engraft in the bone marrow, where they self‐renew potentially for the lifetime of the individual while giving rise to differentiating progeny along all hematopoietic lineages. (
  • 1 Using an animal model of familial hypercholesterolaemia, the Watanabe heritable hyperlipidaemic (WHHL) rabbit, investigators have been able to show the successful transduction of a functional rabbit LDL gene into target hepatocytes. (
  • These evolutionary ancestors of man (Homo Sapiens) had survived bacterial and viral pathogens for 250 million years. (
  • several glycoprotein fragments Below sorry from the former patterns of symptom, treatment, and governmental red brain that the nuclear tobacco to be it that is to cause the bacterial small neuroscientist for criteria. (