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.Gene Library: A large collection of DNA fragments cloned (CLONING, MOLECULAR) from a given organism, tissue, organ, or cell type. It may contain complete genomic sequences (GENOMIC LIBRARY) or complementary DNA sequences, the latter being formed from messenger RNA and lacking intron sequences.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.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.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.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.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).Hematopoietic Stem Cells: Progenitor cells from which all blood cells derive.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.Adenosine Deaminase: An enzyme that catalyzes the hydrolysis of ADENOSINE to INOSINE with the elimination of AMMONIA.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).Transgenes: Genes that are introduced into an organism using GENE TRANSFER TECHNIQUES.Hematopoietic Stem Cell Transplantation: Transfer of HEMATOPOIETIC STEM CELLS from BONE MARROW or BLOOD between individuals within the same species (TRANSPLANTATION, HOMOLOGOUS) or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). Hematopoietic stem cell transplantation has been used as an alternative to BONE MARROW TRANSPLANTATION in the treatment of a variety of neoplasms.Antigens, CD34: Glycoproteins found on immature hematopoietic cells and endothelial cells. They are the only molecules to date whose expression within the blood system is restricted to a small number of progenitor cells in the bone marrow.Cloning, Molecular: The insertion of recombinant DNA molecules from prokaryotic and/or eukaryotic sources into a replicating vehicle, such as a plasmid or virus vector, and the introduction of the resultant hybrid molecules into recipient cells without altering the viability of those cells.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.Libraries, MedicalBase Sequence: The sequence of PURINES and PYRIMIDINES in nucleic acids and polynucleotides. It is also called nucleotide sequence.Libraries: Collections of systematically acquired and organized information resources, and usually providing assistance to users. (ERIC Thesaurus, http://www.eric.ed.gov/ accessed 2/1/2008)Hospitals, University: Hospitals maintained by a university for the teaching of medical students, postgraduate training programs, and clinical research.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.Sequence Analysis, DNA: A multistage process that includes cloning, physical mapping, subcloning, determination of the DNA SEQUENCE, and information analysis.RNA, Ribosomal, 16S: Constituent of 30S subunit prokaryotic ribosomes containing 1600 nucleotides and 21 proteins. 16S rRNA is involved in initiation of polypeptide synthesis.Methanobrevibacter: A genus of gram-positive, anaerobic, cocci to short rod-shaped ARCHAEA, in the family METHANOBACTERIACEAE, order METHANOBACTERIALES. They are found in the GASTROINTESTINAL TRACT or other anoxic environments.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.Combinatorial Chemistry Techniques: A technology, in which sets of reactions for solution or solid-phase synthesis, is used to create molecular libraries for analysis of compounds on a large scale.Genes, Bacterial: The functional hereditary units of BACTERIA.Library Services: Services offered to the library user. They include reference and circulation.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.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.Phylogeny: The relationships of groups of organisms as reflected by their genetic makeup.Libraries, Hospital: Information centers primarily serving the needs of hospital medical staff and sometimes also providing patient education and other services.Bacteria: One of the three domains of life (the others being Eukarya and ARCHAEA), also called Eubacteria. They are unicellular prokaryotic microorganisms which generally possess rigid cell walls, multiply by cell division, and exhibit three principal forms: round or coccal, rodlike or bacillary, and spiral or spirochetal. Bacteria can be classified by their response to OXYGEN: aerobic, anaerobic, or facultatively anaerobic; by the mode by which they obtain their energy: chemotrophy (via chemical reaction) or PHOTOTROPHY (via light reaction); for chemotrophs by their source of chemical energy: CHEMOLITHOTROPHY (from inorganic compounds) or chemoorganotrophy (from organic compounds); and by their source for CARBON; NITROGEN; etc.; HETEROTROPHY (from organic sources) or AUTOTROPHY (from CARBON DIOXIDE). They can also be classified by whether or not they stain (based on the structure of their CELL WALLS) with CRYSTAL VIOLET dye: gram-negative or gram-positive.DNA, Bacterial: Deoxyribonucleic acid that makes up the genetic material of bacteria.Biodiversity: The variety of all native living organisms and their various forms and interrelationships.Genes: A category of nucleic acid sequences that function as units of heredity and which code for the basic instructions for the development, reproduction, and maintenance of organisms.Directed Molecular Evolution: The techniques used to produce molecules exhibiting properties that conform to the demands of the experimenter. These techniques combine methods of generating structural changes with methods of selection. They are also used to examine proposed mechanisms of evolution under in vitro selection conditions.Gene Expression: The phenotypic manifestation of a gene or genes by the processes of GENETIC TRANSCRIPTION and GENETIC TRANSLATION.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.Genomic Library: A form of GENE LIBRARY containing the complete DNA sequences present in the genome of a given organism. It contrasts with a cDNA library which contains only sequences utilized in protein coding (lacking introns).Escherichia coli: A species of gram-negative, facultatively anaerobic, rod-shaped bacteria (GRAM-NEGATIVE FACULTATIVELY ANAEROBIC RODS) commonly found in the lower part of the intestine of warm-blooded animals. It is usually nonpathogenic, but some strains are known to produce DIARRHEA and pyogenic infections. Pathogenic strains (virotypes) are classified by their specific pathogenic mechanisms such as toxins (ENTEROTOXIGENIC ESCHERICHIA COLI), etc.DNA, Ribosomal: DNA sequences encoding RIBOSOMAL RNA and the segments of DNA separating the individual ribosomal RNA genes, referred to as RIBOSOMAL SPACER DNA.Restriction Mapping: Use of restriction endonucleases to analyze and generate a physical map of genomes, genes, or other segments of DNA.Gene Expression Profiling: The determination of the pattern of genes expressed at the level of GENETIC TRANSCRIPTION, under specific circumstances or in a specific cell.National Library of Medicine (U.S.): An agency of the NATIONAL INSTITUTES OF HEALTH concerned with overall planning, promoting, and administering programs pertaining to advancement of medical and related sciences. Major activities of this institute include the collection, dissemination, and exchange of information important to the progress of medicine and health, research in medical informatics and support for medical library development.Biota: The spectrum of different living organisms inhabiting a particular region, habitat, or biotope.Proteobacteria: A phylum of bacteria consisting of the purple bacteria and their relatives which form a branch of the eubacterial tree. This group of predominantly gram-negative bacteria is classified based on homology of equivalent nucleotide sequences of 16S ribosomal RNA or by hybridization of ribosomal RNA or DNA with 16S and 23S ribosomal RNA.Cosmids: Plasmids containing at least one cos (cohesive-end site) of PHAGE LAMBDA. They are used as cloning vehicles.Library Surveys: Collection and analysis of data pertaining to operations of a particular library, library system, or group of independent libraries, with recommendations for improvement and/or ordered plans for further development.Nucleic Acid Hybridization: Widely used technique which exploits the ability of complementary sequences in single-stranded DNAs or RNAs to pair with each other to form a double helix. Hybridization can take place between two complimentary DNA sequences, between a single-stranded DNA and a complementary RNA, or between two RNA sequences. The technique is used to detect and isolate specific sequences, measure homology, or define other characteristics of one or both strands. (Kendrew, Encyclopedia of Molecular Biology, 1994, p503)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).DNA Restriction Enzymes: Enzymes that are part of the restriction-modification systems. They catalyze the endonucleolytic cleavage of DNA sequences which lack the species-specific methylation pattern in the host cell's DNA. Cleavage yields random or specific double-stranded fragments with terminal 5'-phosphates. The function of restriction enzymes is to destroy any foreign DNA that invades the host cell. Most have been studied in bacterial systems, but a few have been found in eukaryotic organisms. They are also used as tools for the systematic dissection and mapping of chromosomes, in the determination of base sequences of DNAs, and have made it possible to splice and recombine genes from one organism into the genome of another. EC 3.21.1.Sequence Homology, Nucleic Acid: The sequential correspondence of nucleotides in one nucleic acid molecule with those of another nucleic acid molecule. Sequence homology is an indication of the genetic relatedness of different organisms and gene function.Library Administration: Planning, organizing, staffing, direction, and control of libraries.Genes, Transgenic, Suicide: Genes that are used transgenically, i.e., via GENE TRANSFER TECHNIQUES to induce CELL DEATH.Sequence Homology, Amino Acid: The degree of similarity between sequences of amino acids. This information is useful for the analyzing genetic relatedness of proteins and species.Soil Microbiology: The presence of bacteria, viruses, and fungi in the soil. This term is not restricted to pathogenic organisms.RNA, Archaeal: Ribonucleic acid in archaea having regulatory and catalytic roles as well as involvement in protein synthesis.Archaea: One of the three domains of life (the others being BACTERIA and Eukarya), formerly called Archaebacteria under the taxon Bacteria, but now considered separate and distinct. They are characterized by: (1) the presence of characteristic tRNAs and ribosomal RNAs; (2) the absence of peptidoglycan cell walls; (3) the presence of ether-linked lipids built from branched-chain subunits; and (4) their occurrence in unusual habitats. While archaea resemble bacteria in morphology and genomic organization, they resemble eukarya in their method of genomic replication. The domain contains at least four kingdoms: CRENARCHAEOTA; EURYARCHAEOTA; NANOARCHAEOTA; and KORARCHAEOTA.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.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.DNA, Archaeal: Deoxyribonucleic acid that makes up the genetic material of archaea.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.Library Science: Study of the principles and practices of library administration and services.Small Molecule Libraries: Large collections of small molecules (molecular weight about 600 or less), of similar or diverse nature which are used for high-throughput screening analysis of the gene function, protein interaction, cellular processing, biochemical pathways, or other chemical interactions.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 2.7.1.21.Genes, rRNA: Genes, found in both prokaryotes and eukaryotes, which are transcribed to produce the RNA which is incorporated into RIBOSOMES. Prokaryotic rRNA genes are usually found in OPERONS dispersed throughout the GENOME, whereas eukaryotic rRNA genes are clustered, multicistronic transcriptional units.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.Libraries, Digital: Libraries in which a major proportion of the resources are available in machine-readable format, rather than on paper or MICROFORM.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.Cell Line: Established cell cultures that have the potential to propagate indefinitely.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.DNA, Complementary: Single-stranded complementary DNA synthesized from an RNA template by the action of RNA-dependent DNA polymerase. cDNA (i.e., complementary DNA, not circular DNA, not C-DNA) is used in a variety of molecular cloning experiments as well as serving as a specific hybridization probe.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.Libraries, NursingGenetic Complementation Test: A test used to determine whether or not complementation (compensation in the form of dominance) will occur in a cell with a given mutant phenotype when another mutant genome, encoding the same mutant phenotype, is introduced into that cell.Recombinant Proteins: Proteins prepared by recombinant DNA technology.Cluster Analysis: A set of statistical methods used to group variables or observations into strongly inter-related subgroups. In epidemiology, it may be used to analyze a closely grouped series of events or cases of disease or other health-related phenomenon with well-defined distribution patterns in relation to time or place or both.Oligonucleotide Array Sequence Analysis: Hybridization of a nucleic acid sample to a very large set of OLIGONUCLEOTIDE PROBES, which have been attached individually in columns and rows to a solid support, to determine a BASE SEQUENCE, or to detect variations in a gene sequence, GENE EXPRESSION, or for GENE MAPPING.Blotting, Southern: A method (first developed by E.M. Southern) for detection of DNA that has been electrophoretically separated and immobilized by blotting on nitrocellulose or other type of paper or nylon membrane followed by hybridization with labeled NUCLEIC ACID PROBES.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.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.Catalogs, LibraryBacterial Proteins: Proteins found in any species of bacterium.Library AssociationsLibrary Collection Development: Development of a library collection, including the determination and coordination of selection policy, assessment of needs of users and potential users, collection use studies, collection evaluation, identification of collection needs, selection of materials, planning for resource sharing, collection maintenance and weeding, and budgeting.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.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.RNA, Bacterial: Ribonucleic acid in bacteria having regulatory and catalytic roles as well as involvement in protein synthesis.Multigene Family: A set of genes descended by duplication and variation from some ancestral gene. Such genes may be clustered together on the same chromosome or dispersed on different chromosomes. Examples of multigene families include those that encode the hemoglobins, immunoglobulins, histocompatibility antigens, actins, tubulins, keratins, collagens, heat shock proteins, salivary glue proteins, chorion proteins, cuticle proteins, yolk proteins, and phaseolins, as well as histones, ribosomal RNA, and transfer RNA genes. The latter three are examples of reiterated genes, where hundreds of identical genes are present in a tandem array. (King & Stanfield, A Dictionary of Genetics, 4th ed)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.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.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.Mice, Inbred C57BLChromosome Mapping: Any method used for determining the location of and relative distances between genes on a chromosome.Time Factors: Elements of limited time intervals, contributing to particular results or situations.Geologic Sediments: A mass of organic or inorganic solid fragmented material, or the solid fragment itself, that comes from the weathering of rock and is carried by, suspended in, or dropped by air, water, or ice. It refers also to a mass that is accumulated by any other natural agent and that forms in layers on the earth's surface, such as sand, gravel, silt, mud, fill, or loess. (McGraw-Hill Dictionary of Scientific and Technical Terms, 4th ed, p1689)Polymorphism, Restriction Fragment Length: Variation occurring within a species in the presence or length of DNA fragment generated by a specific endonuclease at a specific site in the genome. Such variations are generated by mutations that create or abolish recognition sites for these enzymes or change the length of the fragment.Repetitive Sequences, Nucleic Acid: Sequences of DNA or RNA that occur in multiple copies. There are several types: INTERSPERSED REPETITIVE SEQUENCES are copies of transposable elements (DNA TRANSPOSABLE ELEMENTS or RETROELEMENTS) dispersed throughout the genome. TERMINAL REPEAT SEQUENCES flank both ends of another sequence, for example, the long terminal repeats (LTRs) on RETROVIRUSES. Variations may be direct repeats, those occurring in the same direction, or inverted repeats, those opposite to each other in direction. TANDEM REPEAT SEQUENCES are copies which lie adjacent to each other, direct or inverted (INVERTED REPEAT SEQUENCES).Ecosystem: A functional system which includes the organisms of a natural community together with their environment. (McGraw Hill Dictionary of Scientific and Technical Terms, 4th ed)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.Library Technical Services: Acquisition, organization, and preparation of library materials for use, including selection, weeding, cataloging, classification, and preservation.Sequence Alignment: The arrangement of two or more amino acid or base sequences from an organism or organisms in such a way as to align areas of the sequences sharing common properties. The degree of relatedness or homology between the sequences is predicted computationally or statistically based on weights assigned to the elements aligned between the sequences. This in turn can serve as a potential indicator of the genetic relatedness between the organisms.Library Automation: The use of automatic machines or processing devices in libraries. The automation may be applied to library administrative activities, office procedures, and delivery of library services to users.Cell Differentiation: Progressive restriction of the developmental potential and increasing specialization of function that leads to the formation of specialized cells, tissues, and organs.Cell Line, Tumor: A cell line derived from cultured tumor cells.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.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.Students: Individuals enrolled in a school or formal educational program.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.Cumulus Cells: The granulosa cells of the cumulus oophorus which surround the OVUM in the GRAAFIAN FOLLICLE. At OVULATION they are extruded with OVUM.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.Seawater: The salinated water of OCEANS AND SEAS that provides habitat for marine organisms.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.DNA, Fungal: Deoxyribonucleic acid that makes up the genetic material of fungi.Codon: A set of three nucleotides in a protein coding sequence that specifies individual amino acids or a termination signal (CODON, TERMINATOR). Most codons are universal, but some organisms do not produce the transfer RNAs (RNA, TRANSFER) complementary to all codons. These codons are referred to as unassigned codons (CODONS, NONSENSE).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.Library Materials: Print and non-print materials collected, processed, and stored by libraries. They comprise books, periodicals, pamphlets, reports, microforms, maps, manuscripts, motion pictures, and all other forms of audiovisual records. (Harrod, The Librarians' Glossary, 4th ed, p497)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.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.Growth Differentiation Factor 3: A growth differentiation factor that may play a role in maintaining the undifferentiated state of PLURIPOTENT STEM CELLS by inhibiting the actions of BONE MORPHOGENETIC PROTEINS. Differentiation factor 3 is also considered a nodal signaling ligand that influences the genesis of left-right asymmetry.Mice, Inbred BALB CRecombinant 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.Transcription Factors: Endogenous substances, usually proteins, which are effective in the initiation, stimulation, or termination of the genetic transcription process.Genetic Variation: Genotypic differences observed among individuals in a population.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.Flucytosine: A fluorinated cytosine analog that is used as an antifungal agent.Schools, Medical: Educational institutions for individuals specializing in the field of medicine.Gene Expression Regulation, Bacterial: Any of the processes by which cytoplasmic or intercellular factors influence the differential control of gene action in bacteria.Organ Specificity: Characteristic restricted to a particular organ of the body, such as a cell type, metabolic response or expression of a particular protein or antigen.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.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.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.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)Nucleoside Deaminases: Catalyze the hydrolysis of nucleosides with the elimination of ammonia.Facility Design and Construction: Architecture, exterior and interior design, and construction of facilities other than hospitals, e.g., dental schools, medical schools, ambulatory care clinics, and specified units of health care facilities. The concept also includes architecture, design, and construction of specialized contained, controlled, or closed research environments including those of space labs and stations.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)Interlibrary LoansCell Proliferation: All of the processes involved in increasing CELL NUMBER including CELL DIVISION.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.Cell Lineage: The developmental history of specific differentiated cell types as traced back to the original STEM CELLS in the embryo.Research: Critical and exhaustive investigation or experimentation, having for its aim the discovery of new facts and their correct interpretation, the revision of accepted conclusions, theories, or laws in the light of newly discovered facts, or the practical application of such new or revised conclusions, theories, or laws. (Webster, 3d ed)Mice, Knockout: Strains of mice in which certain GENES of their GENOMES have been disrupted, or "knocked-out". To produce knockouts, using RECOMBINANT DNA technology, the normal DNA sequence of the gene being studied is altered to prevent synthesis of a normal gene product. Cloned cells in which this DNA alteration is successful are then injected into mouse EMBRYOS to produce chimeric mice. The chimeric mice are then bred to yield a strain in which all the cells of the mouse contain the disrupted gene. Knockout mice are used as EXPERIMENTAL ANIMAL MODELS for diseases (DISEASE MODELS, ANIMAL) and to clarify the functions of the genes.Mice, Transgenic: Laboratory mice that have been produced from a genetically manipulated EGG or EMBRYO, MAMMALIAN.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.Immunohistochemistry: Histochemical localization of immunoreactive substances using labeled antibodies as reagents.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.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)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).Theileria annulata: A protozoan parasite causing tropical theileriasis in cattle. It is transmitted by ticks of the Hyalomma genus.cis-trans-Isomerases: Enzymes that catalyze the rearrangement of geometry about double bonds. EC 5.2.Libraries, DentalBlotting, Northern: Detection of RNA that has been electrophoretically separated and immobilized by blotting on nitrocellulose or other type of paper or nylon membrane followed by hybridization with labeled NUCLEIC ACID PROBES.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.Cell Division: The fission of a CELL. It includes CYTOKINESIS, when the CYTOPLASM of a cell is divided, and CELL NUCLEUS DIVISION.Phenotype: The outward appearance of the individual. It is the product of interactions between genes, and between the GENOTYPE and the environment.Neoplasm Transplantation: Experimental transplantation of neoplasms in laboratory animals for research purposes.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.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.Proteins: Linear POLYPEPTIDES that are synthesized on RIBOSOMES and may be further modified, crosslinked, cleaved, or assembled into complex proteins with several subunits. The specific sequence of AMINO ACIDS determines the shape the polypeptide will take, during PROTEIN FOLDING, and the function of the protein.Simplexvirus: A genus of the family HERPESVIRIDAE, subfamily ALPHAHERPESVIRINAE, consisting of herpes simplex-like viruses. The type species is HERPESVIRUS 1, HUMAN.History, 20th Century: Time period from 1901 through 2000 of the common era.Treatment Outcome: Evaluation undertaken to assess the results or consequences of management and procedures used in combating disease in order to determine the efficacy, effectiveness, safety, and practicability of these interventions in individual cases or series.Models, Animal: Non-human animals, selected because of specific characteristics, for use in experimental research, teaching, or testing.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).Cell Line, Transformed: Eukaryotic cell line obtained in a quiescent or stationary phase which undergoes conversion to a state of unregulated growth in culture, resembling an in vitro tumor. It occurs spontaneously or through interaction with viruses, oncogenes, radiation, or drugs/chemicals.Bone Marrow Cells: Cells contained in the bone marrow including fat cells (see ADIPOCYTES); STROMAL CELLS; MEGAKARYOCYTES; and the immediate precursors of most blood cells.Injections, Intralesional: Injections introduced directly into localized lesions.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.Academic Medical Centers: Medical complexes consisting of medical school, hospitals, clinics, libraries, administrative facilities, etc.T-Lymphocytes: Lymphocytes responsible for cell-mediated immunity. Two types have been identified - cytotoxic (T-LYMPHOCYTES, CYTOTOXIC) and helper T-lymphocytes (T-LYMPHOCYTES, HELPER-INDUCER). They are formed when lymphocytes circulate through the THYMUS GLAND and differentiate to thymocytes. When exposed to an antigen, they divide rapidly and produce large numbers of new T cells sensitized to that antigen.Gene Expression Regulation, Developmental: Any of the processes by which nuclear, cytoplasmic, or intercellular factors influence the differential control of gene action during the developmental stages of an organism.Retrospective Studies: Studies used to test etiologic hypotheses in which inferences about an exposure to putative causal factors are derived from data relating to characteristics of persons under study or to events or experiences in their past. The essential feature is that some of the persons under study have the disease or outcome of interest and their characteristics are compared with those of unaffected persons.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.Microarray Analysis: The simultaneous analysis, on a microchip, of multiple samples or targets arranged in an array format.RNA: A polynucleotide consisting essentially of chains with a repeating backbone of phosphate and ribose units to which nitrogenous bases are attached. RNA is unique among biological macromolecules in that it can encode genetic information, serve as an abundant structural component of cells, and also possesses catalytic activity. (Rieger et al., Glossary of Genetics: Classical and Molecular, 5th ed)Librarians: Specialists in the management of a library or the services rendered by a library, bringing professional skills to administration, organization of material and personnel, interpretation of bibliothecal rules, the development and maintenance of the library's collection, and the provision of information services.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.Library Schools: Educational institutions for individuals specializing in the field of library science or information.B-Lymphocytes: Lymphoid cells concerned with humoral immunity. They are short-lived cells resembling bursa-derived lymphocytes of birds in their production of immunoglobulin upon appropriate stimulation.Liver: A large lobed glandular organ in the abdomen of vertebrates that is responsible for detoxification, metabolism, synthesis and storage of various substances.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.Genetic Markers: A phenotypically recognizable genetic trait which can be used to identify a genetic locus, a linkage group, or a recombination event.Octamer Transcription Factor-3: An octamer transcription factor that is expressed primarily in totipotent embryonic STEM CELLS and GERM CELLS and is down-regulated during CELL DIFFERENTIATION.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.Students, Medical: Individuals enrolled in a school of medicine or a formal educational program in medicine.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.Questionnaires: Predetermined sets of questions used to collect data - clinical data, social status, occupational group, etc. The term is often applied to a self-completed survey instrument.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.Membrane Proteins: Proteins which are found in membranes including cellular and intracellular membranes. They consist of two types, peripheral and integral proteins. They include most membrane-associated enzymes, antigenic proteins, transport proteins, and drug, hormone, and lectin receptors.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.Signal Transduction: The intracellular transfer of information (biological activation/inhibition) through a signal pathway. In each signal transduction system, an activation/inhibition signal from a biologically active molecule (hormone, neurotransmitter) is mediated via the coupling of a receptor/enzyme to a second messenger system or to an ion channel. Signal transduction plays an important role in activating cellular functions, cell differentiation, and cell proliferation. Examples of signal transduction systems are the GAMMA-AMINOBUTYRIC ACID-postsynaptic receptor-calcium ion channel system, the receptor-mediated T-cell activation pathway, and the receptor-mediated activation of phospholipases. Those coupled to membrane depolarization or intracellular release of calcium include the receptor-mediated activation of cytotoxic functions in granulocytes and the synaptic potentiation of protein kinase activation. Some signal transduction pathways may be part of larger signal transduction pathways; for example, protein kinase activation is part of the platelet activation signal pathway.Curriculum: A course of study offered by an educational institution.Gene Expression Regulation, Neoplastic: Any of the processes by which nuclear, cytoplasmic, or intercellular factors influence the differential control of gene action in neoplastic tissue.Protein Binding: The process in which substances, either endogenous or exogenous, bind to proteins, peptides, enzymes, protein precursors, or allied compounds. Specific protein-binding measures are often used as assays in diagnostic assessments.Online Systems: Systems where the input data enter the computer directly from the point of origin (usually a terminal or workstation) and/or in which output data are transmitted directly to that terminal point of origin. (Sippl, Computer Dictionary, 4th ed)Architecture as Topic: The art and science of designing buildings and structures. More generally, it is the design of the total built environment, including town planning, urban design, and landscape architecture.Stem Cell Transplantation: The transfer of STEM CELLS from one individual to another within the same species (TRANSPLANTATION, HOMOLOGOUS) or between species (XENOTRANSPLANTATION), or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). The source and location of the stem cells determines their potency or pluripotency to differentiate into various cell types.Fibroblasts: Connective tissue cells which secrete an extracellular matrix rich in collagen and other macromolecules.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.Epithelial Cells: Cells that line the inner and outer surfaces of the body by forming cellular layers (EPITHELIUM) or masses. Epithelial cells lining the SKIN; the MOUTH; the NOSE; and the ANAL CANAL derive from ectoderm; those lining the RESPIRATORY SYSTEM and the DIGESTIVE SYSTEM derive from endoderm; others (CARDIOVASCULAR SYSTEM and LYMPHATIC SYSTEM) derive from mesoderm. Epithelial cells can be classified mainly by cell shape and function into squamous, glandular and transitional epithelial cells.
  • Your Genome from the Wellcome Genome campus explains the first gene therapy trial to treat a condition called severe combined immunodeficiency (SCID) . (medlineplus.gov)
  • The new Cas9 approach could also potentially target and activate sections of the genome made up of genes that are not directly responsible for transcription, and which previously were poorly understood. (harvard.edu)
  • If the cell survived, that means its modified genome somehow made it resistant to the virus-it was now missing a pathway that the virus relied on to replicate and do its dirty work. (technologynetworks.com)
  • The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. (kiwix.org)
  • These are due to the advance of genome-wide association studies from the ability to create and sequence genomic libraries. (wikipedia.org)
  • Below are the steps for creating a genomic library from a large genome. (wikipedia.org)
  • Researchers at the J. Craig Venter Institute made the much-anticipated announcement today that they have created a replicating 'synthetic cell' - a bacterium with its DNA replaced by a 100% prosthetic genome. (nanowerk.com)
  • Mitochondria are the energy powerhouses inside our cells, and they carry their own DNA, separate from our nuclear genome. (npr.org)
  • Today, our nuclear genome contains around 20,000 genes, while a scant 37 genes reside in the mitochondria. (npr.org)
  • Our results suggest that the four paralemmin isoform genes (PALM1, PALM2, PALM3 and PALMD) arose by quadruplication of an ancestral gene in the two early vertebrate genome duplications. (jove.com)
  • The key: replacing their defective hearing genes with functional ones. (labroots.com)
  • He and others have applied these PSMA-targeting aptamers to deliver various therapeutic and imaging packages to prostate cancer cells and tumors. (hopkinsmedicine.org)
  • A second related focus of Dr. Mason's lab is to identify novel tumor-associated antigens and to develop single-chain Fragment variable targeting systems that can be used alone or in concert with cell based therapies to target spontaneous occurring tumors. (upenn.edu)
  • These often multi-drug-resistant cells have the ability to generate new tumors, so it is critically important to develop new approaches to more effective and safer treatment or prevention of breast cancer. (prohealth.com)
  • Then specific drugs that target key genes and mutations can be tested in mice and zebrafish with LMS tumors. (sarcomahelp.org)
  • He explains, 'The PI3K-AKT-mTOR pathway is a biochemical pathway that plays an important role in cell division, in other words - the process that allows tumors to grow. (sarcomahelp.org)
  • In the example you see above, NIH-supported researchers Wonkyu Ju, Mark Ellisman, and their colleagues at the University of California, San Diego, engineered adeno-associated virus serotype 2 (AAV2) to deliver a dummy gene tagged with a fluorescent marker (yellow) into the ganglion cells (blue) of a mouse retina. (nih.gov)
  • An experienced life science attorney, Ms. Mikhail cofounded AskBio in 2001 with Jude Samulski , Ph.D., the first scientist to clone Adeno-Associated Virus (AAV), and Xiao Xiao , Ph.D., who was the first to develop a miniaturized dystrophin gene enabling the advancement of gene therapy for Duchenne Muscular Dystrophy. (prnewswire.com)
  • 2002) Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno‐associated virus‐mediated gene delivery. (els.net)
  • EMERYVILLE, Calif.--( BUSINESS WIRE )--4D Molecular Therapeutics (4DMT), a world-leader in Therapeutic Vector Evolution for adeno-associated virus (AAV) gene therapy vector discovery and product development, announced the appointments of Fred Kamal, PhD, as chief technical officer, and August Moretti as chief financial officer. (businesswire.com)
  • Damage of DNA, hence potentially deleterious mutations, caused by such factors as UV, free radicals, and active transcription, needs to be repaired before these mutations are passed to daughter cells. (sigmaaldrich.com)
  • Mitochondria also still divide and replicate like the bacteria they once were, and that constant replication means that mutations arise 10 to 30 times more often in mitochondrial genes than in the nucleus. (npr.org)
  • The defect is caused by variations ( mutations ) in the LAMP2 gene . (nih.gov)
  • Aminoglycosides can bypass nonsense mutations and are the prototypic agents for translational bypass therapy (TBT). (xmu.edu.cn)
  • Armed with this information, the research team will use functional genomics to determine which genes and mutations are important for LMS survival and which are irrelevant. (sarcomahelp.org)
  • Now, a group of researchers from Ospedale San Raffaele , Milan (Italy) and the Telethon Foundation, in collaboration with pharmaceutical giant GSK, have developed their product "Strimvelis", which is the first gene therapy to use genetically modified stem cells. (georgetown.edu)
  • WEDNESDAY, June 16 (HealthDay News) -- Researchers report they've moved a step closer to treating HIV patients with gene therapy that could potentially one day keep the AIDS-causing virus at bay. (bio-medicine.org)
  • In the new study, researchers genetically manipulated blood cells to resist HIV and inserted them into four HIV-positive patients who had lymphoma, a blood cancer. (bio-medicine.org)
  • In a recent study, a team of researchers led by Alwin Köhler at the Max F. Perutz Laboratories (MFPL) belonging to the University of Vienna and the Medical University of Vienna offer new insights into how nuclear pores are constructed in the nuclear envelope. (news-medical.net)
  • To better understand and improve this potential therapeutic strategy, researchers are gauging the efficiency of gene transfer into the retina via an imaging technique called large-scale mosaic confocal microscopy, which computationally assembles many small, high-resolution images in a way similar to Google Earth. (nih.gov)
  • The researchers also used AAV2 to deliver into the retinas of mice a gene that coded for a mutant version of a protein, called DRP1. (nih.gov)
  • The gene transfer proved successful in rescuing the retinal ganglion cells, and researchers are continuing to pursue this line of study with the aim of translating their discoveries into possible ways of helping humans with glaucoma. (nih.gov)
  • Researchers must overcome many technical challenges before gene therapy will be a practical approach to treating disease. (medlineplus.gov)
  • The findings, reported by Church and his team of researchers at the Wyss Institute for Biologically Inspired Engineering at Harvard University and Harvard Medical School in Nature Methods , show promise that precision gene therapies could be developed to prevent and treat disease on a highly customizable, personalized level, which is crucial given the fact that diseases develop among diverse pathways among genetically-varied individuals. (harvard.edu)
  • Researchers Use Mini Form of Replacement Gene to Delay Photoreceptor Degeneration in Leber Congenital Amaurosis. (liebertpub.com)
  • The researchers then exposed the cells to the H5N1 flu strain, a type of influenza A virus commonly known as the bird flu. (technologynetworks.com)
  • Researchers have devised the first techniques that directly assess "off-target effects" of gene-cutting zinc finger nucleases (ZFNs). (acs.org)
  • Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K. (news-medical.net)
  • Calculating the titer allows researchers to approximate how many infectious viral particles were successfully created in the library. (wikipedia.org)
  • This allows researchers to also determine the percentage of infectious viral particles actually carrying a fragment of the library. (wikipedia.org)
  • By doing this test, researchers can also determine the efficiency of the ligation and make adjustments as needed to ensure they get the desired number of clones for the library. (wikipedia.org)
  • Researchers at the J. Craig Venter Institute published results today describing the successful construction of the first self-replicating, synthetic bacterial cell. (nanowerk.com)
  • In sum, researchers are now being more realistic than in the early days, seeing the future of gene therapy in providing effective treatments, not cures. (sigmaaldrich.com)
  • Researchers used a gene-carrying virus to fix blood stem cells that were then used to treat patients with beta-thalassemia. (npr.org)
  • Some of the patients have been followed for more than three years and so far there have been no signs of any serious side effects from the gene therapy, the researchers reported. (npr.org)
  • While saying the one-time gene therapy was an ideal treatment goal because of its effects, the researchers stopped short of calling it a cure for hemophilia B since it's unclear if the benefits will be permanent. (wlox.com)
  • All of the patients benefited from the gene therapy, the researchers said. (wlox.com)
  • The researchers administered these six at bioavailable levels to both breast cancer and control cells. (prohealth.com)
  • The researchers observed no harmful effects on the control cells. (prohealth.com)
  • The researchers also earlier demonstrated that two of the compounds synergize effectively to kill ovarian cancer cells. (prohealth.com)
  • Researchers have strived to develop cell-based therapies that will not only manage the associated symptoms of disc degeneration but also reverse the degenerative process. (umd.edu)
  • Using a new optical fiber in vivo imaging technique (CellviZio developed by Mauna Kea Technologies of Paris), the UB researchers were able to observe the brain cells expressing genes without having to sacrifice the animal. (medgadget.com)
  • Another interesting recent development is a report by MIT researchers that they have found a way to create gene carriers from biodegradable polymers instead of viral materials . (highlighthealth.com)
  • Researchers chemically modified the ends of the polymer chains using a library of small molecules to attenuate and optimize nanoparticle formation and DNA delivery. (highlighthealth.com)
  • There is significant evidence that inhibition of HIF-1 would be beneficial for cancer therapy, since tumor cells must thrive in a microenvironment characterized by lack of oxygen. (lifeboat.com)
  • When this method is used to deliver the Fas ligand (FasL) gene, some tumor cells are highly susceptible to its death-promoting effects, while others are resistant. (akcchf.org)
  • One question that this project sought to answer was whether there was a relationship between the susceptibility of the tumor cells to FasL-mediated death and the patient's response to therapy. (akcchf.org)
  • On the other hand, certain cancers can exploit a negative regulator like CIC to suppress immune responses while tumor cells run rampant. (technologynetworks.com)
  • AIM: ERC1671 is an allogeneic/autologous therapeutic glioblastoma (GBM) vaccine - composed of whole, inactivated tumor cells mixed with tumor cell lysates derived from the patient and three GBM donors. (wur.nl)
  • Chemokines have been shown to be selective chemo-attractants for leukocyte sub-populations in vitro and to elicit a selective accumulation of immune cells in vivo . (frontiersin.org)
  • The cells can readily be converted into functional hepatocytes in vitro and upon transplantation in vivo. (uu.nl)
  • A portion of cells will be kept as 'back-up' for rescue purpose if necessary, and a portion will be ex vivo gene-modified with a lentiviral vector, pCCL-CTNS, to express CTNS gene (product name: CTNS-RD-04). (clinicaltrials.gov)
  • The results indicated that circAGFG1 could promote TNBC cell proliferation, mobility and invasion as well as tumorigenesis and metastasis in vivo. (springer.com)
  • One approach involves active immunization using whole tumor RNA loaded CD40 activated B cells, aimed at activating tumor specific T cells in vivo. (upenn.edu)
  • Pancreatic cancer cells were studied both in vitro and in vivo. (lu.se)
  • Conclusions: AdTPHre-hEndo has a special ability to duplicate and kill pancreatic cancer cells in in vitro and in vivo experiments, thus providing a new gene-virus-based treatment system for pancreatic cancer. (lu.se)
  • The importance of this success contributed to the ever-increasing demand for sequencing genomes to research gene therapy. (wikipedia.org)
  • Teams are now able to catalog polymorphisms in genomes and investigate those candidate genes contributing to maladies such as Parkinson's disease, Alzheimer's disease, multiple sclerosis, rheumatoid arthritis, and Type 1 diabetes. (wikipedia.org)
  • This is especially true of mitochondria, because its genomes are so different than the genomes in the nucleus of cells. (npr.org)
  • We have found evidence for such an arrangement for paralemmin-1 and -2 in several vertebrate genomes, as well as for palmdelphin and paralemmin-3 in teleost fish genomes, and suggest the name paralemmin downstream genes (PDG) for this new gene family. (jove.com)
  • Although only about the size of a postage stamp, the retina contains more than 100 distinct cell types that are organized into multiple information-processing layers. (nih.gov)
  • In people with inherited disorders in which the retina degenerates, an altered gene somewhere within this nexus of cells progressively robs them of their sight. (nih.gov)
  • Gene Therapy: A Breakthrough for Sickle Cell Anemia? (lakelandhealth.org)
  • The disparity in stroke-related deaths among black and white children dramatically narrowed after prevention strategies changed to include ultrasound screening and chronic blood transfusions for children with sickle cell anemia, according to research presented at the American Stroke Association's International Stroke Conference 2012. (news-medical.net)
  • This trial, supported through grants from the National Cancer Institute of the National Institutes of Health and the AKC Canine Health Foundation, was meant to define the safety, efficacy, and mechanisms of Fasaret®, a formulation for the Fas ligand gene expressed in a viral vector and produced by ApopLogic Pharmaceuticals, Inc. (akcchf.org)
  • Intranasal Gene Delivery: Noninvasive Approach Shows Efficacy for Preventing Neurologic Effects of Inherited Metabolic Disease. (liebertpub.com)
  • Promising Safety and Efficacy Results With Novel Lentiviral-Based Gene Therapy for SCID. (liebertpub.com)
  • Vaccines have variable efficacy, and the virus has a propensity to mutate so that antiviral drugs don't work as well," said Julianna Han, a graduate student in microbiology at UChicago and lead author of the study, published April 10, 2018 in Cell Reports. (technologynetworks.com)
  • This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (clinicaltrials.gov)
  • While still an experimental therapy, the dangers are now much better understood, and the issues facing companies with gene therapy products have a more defined standard of efficacy and safety. (sigmaaldrich.com)
  • A new approach developed by Harvard geneticist George Church, Ph.D., can help uncover how tandem gene circuits dictate life processes, such as the healthy development of tissue or the triggering of a particular disease, and can also be used for directing precision stem cell differentiation for regenerative medicine and growing organ transplants. (harvard.edu)
  • Clonal long-term expansion of primary adult liver stem cells opens up experimental avenues for disease modeling, toxicology studies, regenerative medicine, and gene therapy. (uu.nl)
  • Using this approach, Dr. Lupold developed the first RNA aptamers targeted to a cancer cell-surface antigen. (hopkinsmedicine.org)
  • Identification and characterization of nuclease-stabilized RNA molecules that bind human prostate cancer cells via the prostate-specific membrane antigen. (hopkinsmedicine.org)
  • If you build a facility for chimeric antigen receptor (CAR) T-cell processing, you will always find somebody who has an interest in using that facility," Niss argues. (genengnews.com)
  • A second approach involves the passive adoptive transfer of genetically modified autologous T cells that are capable of MHC-independent tumor antigen recognition and activaton/effector function in the absence of co-stimulatory ligands. (upenn.edu)
  • RNA-loaded CD40-activated B cells stimulate antigen-specific T-cell responses in dogs with spontaneous lymphoma. (upenn.edu)
  • After extensive research on animals throughout the 1980s and a 1989 bacterial gene tagging trial on humans, the first gene therapy widely accepted as a success was demonstrated in a trial that started on 14 September 1990, when Ashi DeSilva was treated for ADA - SCID . (kiwix.org)
  • 2002) Correction of ADA‐SCID by stem cell gene therapy combined with nonmyeloablative conditioning. (els.net)
  • A key advantage of the UB team's nanoparticle is its surface functionality, which allows it to be targeted to specific cells, explained Dhruba J. Bharali, Ph.D., a co-author on the paper and post-doctoral associate in the UB Department of Chemistry and UB's Institute for Lasers, Photonics and Biophotonics. (medgadget.com)
  • Current commercial PCRs tests for identifying Salmonella target genes unique to this genus. (jove.com)
  • The results, which also revealed potential treatment target genes, are published in the November 2013 issue of The Journal of Cancer. (prohealth.com)
  • Feng Zhang Discusses CRISPR and Future of Gene Editing in Interview in Human Gene Therapy. (liebertpub.com)
  • Dr. Batra discussed MilliporeSigma's efforts to advance CAR-T therapy production, as well as its CRISPR business and some recent activity in biomanufacturing, in an interview conducted this week during the J.P. Morgan 36th Annual Healthcare Conference, held here at the Westin St. Francis Hotel. (genengnews.com)
  • In this work, promiscuous chemokine-binding peptides that could bind and inhibit multiple inflammatory chemokines, such as CCL2, CCL5, and CXCL9/10/11, were selected from phage display libraries. (frontiersin.org)
  • One area of interest is high-diversity polymer libraries, including aptamers and peptides, which can be screened to identify high-affinity cancer ligands. (hopkinsmedicine.org)
  • In the study published in the journal Science Signaling, the team led by LLuís Espinosa, investigator of IMIM's research group into stem cells and cancer, have shown that inhibition of endosomal activity is a potential therapeutic strategy for the treatment of cancers with the BRAF mutated gene. (news-medical.net)
  • Gene therapy techniques were used to insert a peptide into cultures of human cancer cells that blocked their ability to use the enzyme Hypoxia-inducible factor-1, a heterodimeric transcription factor that enables cell survival under low oxygen conditions by altering the transcription of over 300 genes. (lifeboat.com)
  • If a virus was found to target cancer cells, this virus could then be used as a treatment. (thefreelibrary.com)
  • Animal Cancer Care and Research (ACCR) members from the University of Minnesota were key participants in a recently completed clinical trial testing a gene therapy approach to treat canine osteosarcoma. (akcchf.org)
  • Moreover, they highlighted mechanisms through which this therapy would improve outcome for bone cancer patients. (akcchf.org)
  • Next steps for this therapy are validation in community-based trials, and possibly its application to other types of cancer. (akcchf.org)
  • Image and caption reprinted from Modiano et al, "Naturally Occurring Translational Models for Development of Cancer Gene Therapy. (akcchf.org)
  • This edition covers the topics found in the first two editions, while updating our growing knowledge of topics such as molecular oncology, intracellular signaling, control of cell proliferation, immunology of cancer, advances in radiotherapy, and many more. (oncolink.org)
  • The drug works by acting on a faulty gene, BRAF, found in half of terminally ill patients whose cancer has spread to other organs. (futuretimeline.net)
  • German Research Advances in Cancer and Blood Disorders Reported in Human Gene Therapy. (liebertpub.com)
  • Cancer therapy is challenging due to multiple reasons and unwanted side effects. (nova.edu)
  • Metabolically cancer cells are very different compared to normal cells. (nova.edu)
  • For example cancer cells live and thrive anaerobically (hypoxic) even when there is plenty of oxygen around. (nova.edu)
  • Therefore, understanding cancer cell metabolism is extremely significant to come up with drug targets that are better than the existing cancer drugs. (nova.edu)
  • We have demonstrated that cancer cell in culture when deprived of key amino acid methionine, undergoes nutrient deprivation induced cell aggregation or cell death. (nova.edu)
  • The severity of cancer cell aggregation or death varies between ovarian, prostate and kidney cell lines. (nova.edu)
  • This sets a strong basis for our current proposal to further understand the amino acid metabolism of prostate cancer cells. (nova.edu)
  • Thus, the fundamental process of DNA and chromatin modeling/remodeling that is quite unique between normal and cancer cells is dependent on methionine/SAM. (nova.edu)
  • There are still no approved gene therapy products in the western world, but numerous applications are being pursued, notably in cancer, central nervous system disorders and cardiac disease. (sigmaaldrich.com)
  • Poly (AT) polymorphism in the XPC gene and smoking enhance the risk of prostate cancer in a low-risk. (biomedsearch.com)
  • Identification of novel cancer genes for molecular therapy and diagnosis is a current focus of breast cancer research. (xmu.edu.cn)
  • CD40-activated B cell cancer vaccine improves second clinical remission and survival in privately owned dogs with non-Hodgkin's lymphoma. (upenn.edu)
  • Princess Margaret Cancer Centre, 610 University Ave, Room 7-605. (utoronto.ca)
  • Background: Gene-virus targeted therapy is a promising new method of treating pancreatic cancer. (lu.se)
  • Duplication and lethal assays proved that AdTPHre-hEndo could replicate specifically in pancreatic cancer cells and kill them. (lu.se)
  • article{4ff1d940-8596-4c8a-a9d8-50badfc222a8, abstract = {Background: Gene-virus targeted therapy is a promising new method of treating pancreatic cancer. (lu.se)
  • New Orleans, LA - A study led by Madhwa Raj, PhD, Research Professor in Obstetrics and Gynecology at LSU Health Sciences Center New Orleans and its Stanley S. Scott Cancer Center, has found that a super cocktail of six natural compounds in vegetables, fruits, spices and plant roots killed 100% of sample breast cancer cells without toxic side effects on normal cells. (prohealth.com)
  • One of the primary causes of both the recurrence of breast cancer and deaths is a small group of cancer stem cells that evade therapy," notes Dr. Raj. (prohealth.com)
  • When combined, though, the super cocktail suppressed breast cancer cell growth by more than 80%, inhibited migration and invasion, caused cell cycle arrest, and triggered the process leading to cell death resulting in the death of 100% of the breast cancer cells in the sample. (prohealth.com)
  • Our group has had great success in using this approach in other sarcomas to define new classes of drugs that kill human cancer cells. (sarcomahelp.org)
  • He then went on to get his doctorate in human genetics from Johns Hopkins University in 1990. (wikipedia.org)
  • A new approach to combatting tuberculosis would take advantage of a complex, natural process called autophagy that the human body uses to recycle nutrients, remove damaged cell components, eliminate invading bacteria, and respond to inflammation. (news-medical.net)
  • With a view to demonstrating the potential for encoding the production of a therapeutic agent in response to a disease marker, the investigators engineered human cells with an additional chromosomal control circuit that conditionally encoded the production of the cyclic peptide HIF-1 inhibitor. (lifeboat.com)
  • Now, a study published in Human Reproduction has proposed treating the condition with adult stem cells obtained from the patient's own menstrual blood. (georgetown.edu)
  • Professor Salih Sanlioglu, chairman of the Human Gene Therapy Division of the Akdeniz University's Department of Medical Genetics, and his team's research rising hopes for patients with Type 1 diabetes made to the cover of October edition of the Human Gene Therapy magazine in the United States. (thefreelibrary.com)
  • Gene Therapy to Prevent Vision Loss: Studies in Primates Needed to Optimize Human Trials. (liebertpub.com)
  • They created a library of modified human epithelial lung cells, the cells that line the airways and are the first to be infected by the flu virus. (technologynetworks.com)
  • We now describe conditions allowing long-term expansion of adult bile duct-derived bipotent progenitor cells from human liver. (uu.nl)
  • The first attempt at modifying human DNA was performed in 1980 by Martin Cline , but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health , was performed in May 1989. (kiwix.org)
  • Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies. (kiwix.org)
  • HSV-1 amplicon-mediated post-transcriptional inhibition of Rad51 sensitizes human glioma cells to ionizing radiation. (uzh.ch)
  • The genes in mitochondria, which are the powerhouses in human cells, can cause fatal inherited disease. (npr.org)
  • Human Gene Therapy 11: 179-190. (els.net)
  • simian-human from the materials and address(es posed above, preventionHow a Bulgarian strategic cells and wrong society weeks are, and we could still sure enhance them not totally. (anthonyflood.com)
  • In the UB experiments, targeted dopamine neurons - which degenerate in Parkinson's disease, for example - took up and expressed a fluorescent marker gene, demonstrating the ability of nanoparticle technology to deliver effectively genes to specific types of cells in the brain. (medgadget.com)
  • The process of producing and delivering the therapies to patients can take 30 to 40 days, relying on cumbersome methods based on older cell expansion technologies repurposed for CAR-T. (genengnews.com)
  • Various methods for separating the multiple cell types of the nucleus pulposus were evaluated, and filtration was chosen as the most acceptable. (umd.edu)
  • Now chemists at the University of Nottingham are using green chemistry to develop new methods for coating drugs in plastics . (highlighthealth.com)
  • Another patient, who had had evidence of disease progression on MRI, had withdrawn from the study to undergo allogeneic stem-cell transplantation and later died from transplantation-related complications. (nih.gov)
  • Early results of this study suggest that Lenti-D gene therapy may be a safe and effective alternative to allogeneic stem-cell transplantation in boys with early-stage cerebral adrenoleukodystrophy. (nih.gov)
  • He's an associate professor of pediatrics-stem cell transplantation at Stanford University School of Medicine. (wlox.com)
  • Laboratory studies have shown that when CD8 T‐cells are modified with SL9 TCRs, they kill cells that are infected with HIV better than normal CD8 T‐cells can. (clinicaltrials.gov)
  • A part of our laboratory develops tools to assay and exploit physical properties of cells in diagnostics and drug screening. (selectbiosciences.com)
  • A University of Michigan Health System laboratory study reveals a key trigger for producing normal red blood cells that could lead to a new treatment for those with sickle cell disease. (news-medical.net)
  • At Mustang Bio, laboratory manager Mohammed Ridha (left) and analytical development scientist Kennedy Okechukwu (right) work in the company's CAR T-cell therapy manufacturing facility in Worcester, MA. (genengnews.com)
  • Vesicle traffic acts as a kind of "home-delivery service" in cells. (news-medical.net)
  • Gene delivery relies upon the encapsulation of a gene of interest, which is then ideally delivered to target cells. (uwaterloo.ca)
  • Two months after AAV-mediated gene delivery, yellow had overlaid most of the blue, indicating the dummy gene had been selectively transferred into retinal ganglion cells at a high rate of efficiency . (nih.gov)
  • Efficient delivery of the desired gene and substantial release inside the cell is the major hurdle in gene therapy," explained Dhruba J. Bharali, Ph.D., a co-author and postdoctoral researcher in the UB Department of Chemistry and UB's Institute for Lasers, Photonics and Biophotonics, where the work was done. (buffalo.edu)
  • No gene-delivery vehicle -- either viral or non-viral -- has ever been tracked in the cell before," explained Tymish Y. Ohulchanskyy, Ph.D., the third co-author and post-doctoral research scholar at the institute. (buffalo.edu)
  • By using our photonics approach, we can track gene delivery step by step to optimize efficiency," he said. (buffalo.edu)
  • New Gene Delivery Approach Could Allow Long-Term Persistence in Proliferating Cells. (liebertpub.com)
  • New Approach to Improve Therapeutic Gene Delivery to Arthritic Joints. (liebertpub.com)
  • Gene Delivery to the Lung Can Treat Broad Range of Diseases Within and Beyond the Lung. (liebertpub.com)
  • The Better Health Channel from the State Government of Victoria (Australia) provides a brief introduction to gene therapy , including the gene therapy process and delivery techniques. (nih.gov)
  • The outstanding achievement allows Professor Azzouz and his team to expand the horizon of translational research at SITraN and develop tools for efficient, safe and selective delivery of therapies to the central nervous system (CNS) to treat neurological disorders. (sheffield.ac.uk)
  • In participation with Blog Action Day , an event where bloggers from around the world unite to put a single important issue on everyone's mind - the environment - today's article discusses recent advances in the use of biodegradable materials for drug and gene delivery. (highlighthealth.com)
  • Katherine High Discusses Progress and Challenges in Gene Therapy for Hemophilia and Inherited Retinopathies. (liebertpub.com)
  • HealthDay News) -- Gene therapy has helped 10 men with a form of the bleeding disorder hemophilia produce a critical blood clotting factor. (wlox.com)
  • Hemophilia arises from an inherited gene mutation that impedes the ability to produce normal levels of a blood clotting factor. (wlox.com)
  • For the study, George and her colleagues delivered one dose of the gene therapy each into 10 hemophilia B patients' livers. (wlox.com)
  • Another hurdle is that about one-third of hemophilia B patients have a pre-existing immunity to the virus that delivers this type of gene therapy, making them ineligible to receive it, George and Porteus noted. (wlox.com)
  • We have shown that using photonics, the gene-therapy transfer can be monitored, tracking how the nanoparticle penetrates the cell and releases its DNA in the nucleus," explained Paras N. Prasad, Ph.D., executive director of the UB Institute for Lasers, Photonics and Biophotonics, SUNY Distinguished Professor in the Department of Chemistry in the University at Buffalo's College of Arts and Sciences, and a co-author of the paper. (buffalo.edu)
  • The bacteria eventually evolved into mitochondria, migrating most of their genes to the cell nucleus and keeping just a few dozen, largely to help them produce energy. (npr.org)
  • The Harvard team focused on a specific gene involved in hearing, TMC1. (labroots.com)
  • The work could lead to ZFNs that more precisely target specific genes for use in gene therapy or as research tools. (acs.org)
  • Analysis of allergen-specific antibody titers revealed that all animals produced allergen-specific antibodies, and allergen-specific stimulation of CD4 T-cells revealed a significant higher percentage of IFN¿-expressing CD4 T-cells in healthy ponies compared to IBH-affected ponies. (wur.nl)
  • In order to construct a genomic library, the organism's DNA is extracted from cells and then digested with a restriction enzyme to cut the DNA into fragments of a specific size. (wikipedia.org)
  • Our approach of MGLD gene therapy to dwindle methionine nutrient pool is quite unique, novel and we hope our understanding of methylation metabolism will eventually lead to better and specific drug target and dietary strategies for cancers. (nova.edu)
  • This allows us to conclude that the paralemmin gene family arose early and has been maintained throughout vertebrate evolution, suggesting functional diversification and specific biological roles of the paralemmin isoforms. (jove.com)
  • The paralemmin genes have also maintained specific features of gene organisation and sequence. (jove.com)
  • We are building out the next phase in a more cell processing-specific way. (genengnews.com)
  • The PCR primer pairs were targeted to key genes or sequences unique to a specific Salmonella serovar and designed to produce an amplicon with size specific for that gene or allele. (jove.com)
  • Salmonella serovar is assigned to an isolate based on the combination of PCR test results for specific LPS and flagellin gene alleles. (jove.com)
  • They generated a virus library containing about 200,000 variants and identified capsid changes that both maintained AAV2's viability and improved its "homing" potential (tropism) to specific organs in mice. (technologynetworks.com)