Novel gene therapy viral vector mediated rna interference rna viral. Medical search. Frequent questions

Natasha Jane Caplen is a British-American geneticist who discovered RNA interference (RNAi) in mammalian cells. (wikipedia.org)
It was while at NHGRI that Caplen developed a research interest in the newly identified gene silencing mechanism, RNA interference (RNAi) leading to her studies that establish the presence of RNAi in mammalian cells. (wikipedia.org)
these agents act through the natural RNA interference (RNAi) pathway. (chi-peptalk.com)
Our laboratory demonstrated the very first in vivo RNAi mediated silencing in mouse liver and jejunum in 2004.To deliver therapeutic siRNAs into liver hepatocytes, we have further developed a three-pronged approach with the goals of enabling delivery to hepatocytes after both intravenous and subcutaneous administration. (chi-peptalk.com)
To address this challenge I, and others, developed experimental strategies for the discovery and elucidation of gene function by exploiting the RNA interference (RNAi) gene silencing mechanism to generate loss-of-function (LOF) phenotypes. (nih.gov)
My research interests build on my experience in the nucleic acid delivery/gene therapy research field, my work on the RNAi mechanism (including studies establishing the existence of RNAi in mammalian cells), and my independent and collaborative endeavors developing RNAi based analysis and screening approaches for the study of gene function. (nih.gov)
My laboratory uses functional genetic approaches induced by RNAi and other DNA or RNA-based technologies to interrogate specific aspects of the genetic, transcriptional, and cell-signaling alterations observed in cancer cells. (nih.gov)
RNA interference (RNAi) has been regarded as a vital asset in the field of therapeutics as it has the capability to silence various disease causing genes including those that cause cancer. (nih.gov)
Small non-coding RNA molecules such as short interfering RNAs (siRNAs) are one of the extensively studied RNAi inducers for gene modulations. (nih.gov)
Corbin L (2023) RNA Interference (RNAi) Therapies for Rare Genetic Disorders. (scitechnol.com)
The advent of RNA interference (RNAi) technology offers new hope in addressing the root cause of these genetic disorders. (scitechnol.com)
RNAi therapies can specifically silence disease-causing genes, potentially halting disease progression and improving patients' quality of life. (scitechnol.com)
RNAi is a natural cellular process that regulates gene expression through the degradation or translational inhibition of specific messenger RNA (mRNA) molecules. (scitechnol.com)
The specificity and versatility of RNAi make it an attractive therapeutic approach for genetic disorders caused by single gene mutations. (scitechnol.com)
By delivering synthetic siRNAs or miRNAs that target disease-causing genes, RNAi therapies can effectively silence the expression of faulty proteins, correcting the underlying molecular defect. (scitechnol.com)
Over the past decade, RNAi technology has witnessed remarkable progress, leading to the development of several RNAi-based therapies approved by regulatory agencies. (scitechnol.com)
Patisiran is an RNAi therapeutic that targets and silences the Transthyretin (TTR) gene in hATTR patients, preventing the accumulation of amyloid deposits in tissues. (scitechnol.com)
These approved RNAi therapies have demonstrated not only impressive efficacy but also a favorable safety profile, validating the potential of RNAi technology as a viable treatment option for rare genetic disorders. (scitechnol.com)
In DMD, for instance, RNAi therapies targeting the dystrophin gene could hold the key to restoring functional dystrophin expression in muscle cells. (scitechnol.com)
Ongoing research is focused on optimizing the delivery and efficacy of RNAi therapies to further enhance outcomes for SMA patients of all ages. (scitechnol.com)
Despite the considerable progress made in RNAi therapies, challenges remain in their widespread implementation for rare genetic disorders. (scitechnol.com)
RNA interference (RNAi) has been considered a significant asset within the area of therapeutics because it has the potential to silence varied illness inflicting genes together with people who trigger most cancers. (lcabie.com)
Our approach utilizes PTD-mediated delivery of small double stranded RNA molecules, called siRNAs, that induce a RNA interference or RNAi response that selectively eliminates expression of specific gene products. (ca.gov)
Recent advances in research and science have enabled the investigation of a new potential treatment involving gene-based therapy, known as RNA interference (RNAi) that will direct gene silencing and further compensate for natural variants and viral mutants. (springeropen.com)
This paper examines the mechanism of RNAi as a viable HIV therapy, using a minimum of four shRNAs to target both dispensable host components (CCR5) and viral genes (Gag, Env, Tat, Pol I, Pol II and Vif). (springeropen.com)
One of the prime example is gene therapy approach that uses RNA interference (RNAi) to silence viral expression or host mRNA targets essential for HIV-1 infection and replication [ 3 ]. (springeropen.com)
The primary advantage of utilizing RNAi is that it compensates for viral mutants as well as natural variants, thus increasing the quantity of therapeutic targets way more than the capabilities possessed by cART [ 4 ]. (springeropen.com)
Therefore, this review highlights the use of RNAi-mediated silencing as a treatment for HIV-1 infection. (springeropen.com)
S ince its discovery 16 years ago, researchers have been eyeing RNA interference (RNAi)-a natural process of posttranscriptional silencing of genes by small fragments of the nucleic acid-for its potential in therapy, especially in treating forms of cancer and other diseases that are particularly hard to address with existing drugs. (the-scientist.com)
But the path of such RNAi therapies to the clinic has been nothing short of a pharmaceutical roller-coaster ride. (the-scientist.com)
At the same time, the scientific community began to develop a growing interest in RNAi therapies. (the-scientist.com)
Major pharmaceutical companies invested several billion dollars in RNAi therapeutics "regardless of technical obstacles," says Haussecker, who specializes in RNAi and other RNA-based therapies. (the-scientist.com)
While RNAi is considered important in most viral infections, many other pathways can exist that confer antiviral resistance. (preprints.org)
In several cases, protection against viral infection can be obtained in RNAi mutants by non-RNAi mechanisms, confirming the variability of the RNAi defense mechanism according to the type of infection and the physiological status of the host. (preprints.org)
While Drosophila can function as a useful model, this issue may be more critical for economically important insects that are either controlled (agricultural pests and vectors of diseases) or protected from parasite infection (beneficial insects as bees) by RNAi products. (preprints.org)
Recent intense work in the field has shown that small dsRNAs mediate sequence specific RNA degradation in the process known as RNA interference (RNAi). (nih.gov)
November 2013 - Scientists at NIH used RNA interference (RNAi) technology to reveal dozens of genes that may represent new therapeutic targets for treating Parkinson's disease . (nih.gov)

Small interfering RNAs (siRNAs) can be targeted to tumors and one example is the suppression of H-ras gene expression indicating the potential for application in therapy of ovarian cancer. (researchandmarkets.com)
This process is initiated by small RNA molecules, including Small Interfering RNAs (siRNAs) and microRNAs (miRNAs), which guide the RNA-Induced Silencing Complex (RISC) to target mRNAs. (scitechnol.com)
Over the last 9 months of funding, we have rapidly advanced this approach, called PTD-DRBD mediated siRNA delivery, and find that it is non-cytotoxic, delivers siRNAs into the entire population of all cell types, including stem cells and iPS cells, and has a minimal effect on the overall cell biology or other non-targeted gene expression profiles. (ca.gov)
When V. dahliae protoplasts were transformed with short interfering RNAs (siRNAs) targeting the VdAAC gene, fungal growth and sporulation were significantly inhibited. (preprints.org)

MicroRNAs (miRNAs) are endogenous 21-23 nt small non-coding RNA molecules that binding to a multiple of target mRNAs and functioning in the regulation of viral replication including the miRNA-mediated antiviral defense. (biomedcentral.com)
9-10) His major accomplishment is his invention of a new class of gene silencing molecules called small interfering RNA (siRNA). (truebluevision.ca)
In this review, 'specificity' is defined as the ability of an OGT agent to bind only a targeted RNA sequence in a complex mixture of biological molecules, such as those found in human body, without interacting with other biomolecules including non-targeted RNAs. (thno.org)

[ 77-79 ] These diseases are particularly amenable to gene therapy as they can be addressed through easily accessible stem cell populations (hematopoietic) that can be transduced ex vivo (allowing for greater transduction efficiency) with a single gene and expanded in vivo . (medscape.com)
Subsequent gene delivery could be used to enhance, direct and sustain NSC populations through direct in vivo or ex vivo transduction with genes that may affect these outcomes. (medscape.com)
Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters. (neurotree.org)
16. Viral vector-mediated transduction of a modified thrombospondin-2 cDNA inhibits tumor growth and angiogenesis. (nih.gov)
The tissue tropism of AAV4 vector has been validated in lung and heart, as well as low transduction efficiency and viral genome copy numbers in the liver, with potential applications in tissue-specific gene therapy. (genemedi.net)
Our labs have developed small domains from proteins called cell-permeable peptides or peptide transduction domains (PTDs) that enter cells, including embryonic stem cells and non-dividing adult stem cells, in a non-cytotoxic manner that is independent of exposing the stem cells to DNA vectors. (ca.gov)

Recombinant adeno-associated viral vectors are an important technology for novel in vivo gene therapeutics. (chi-peptalk.com)

Discover analytical workflows for product and process related impurities including viral capsid purity, genome integrity and empty/full vector ratio assessment. (chi-peptalk.com)
Technological advances have revolutionized the genome-scale profiling of DNA copy number and sequence, DNA and chromatin modification, and gene expression and have substantially enhanced our understanding of the molecular changes that underlie many cancers. (nih.gov)
Intratumoral administration of MVA induces a cyclic GMP-AMP synthase-mediated kind I interferon (IFN) response and the manufacturing of excessive ranges of the transgenes engineered into the viral genome equivalent to tumor antigens to assemble most cancers vaccines. (lcabie.com)
The chairs of the workshop I want to thank are Drs. Mustafa Sahin from Boston Children's Hospital, Guangping Gao from the University of Massachusetts, Sitra Tauscher-Wisniewski from Novartis Gene Therapies, Beverly Davidson from Children's Hospital of Philadelphia, Cynthia Tifft from National Human Genome Research Institute and Terrance Flotte from University of Massachusetts. (nih.gov)
Limouse C, Jukam D, Smith OK, Fryer KA and Straight AF, 2020, Mapping Transcriptome-Wide and Genome-Wide RNA-DNA Contacts with Chromatin-Associated RNA Sequencing (ChAR-seq). (stanford.edu)
Multivariate survival analysis was performed for the prognostic genes, and the GBM data set from The Cancer Genome Atlas database was utilized to validate the prognostic genes. (cancerindex.org)
The active n-butanol fraction was evaluated for its inhibition against HIV-1 reverse transcriptase, integrase, protease, pro-viral genome integration and viral Tat protein mediated transactivation. (biomedcentral.com)
The provirus integrated in the host genome may remain in a quiescent state in the resting lymphocytes until basal transcription produces a threshold level of the viral trans-activator protein, Tat. (biomedcentral.com)
12. Lloyd A, Vickery ON, Laugel B. Beyond the antigen receptor: editing the genome of T-cells for cancer adoptive cellular therapies. (ac.ir)

Everyone, I welcome you all to the NIMH virtual workshop on gene based therapeutics for genetic neurodevelopmental disorders. (nih.gov)
Today we have a panel of experts from academia, government and industry to share some success stories and their perspectives on challenges and opportunities in developing gene based therapeutics for individuals with rare genetic neurodevelopmental disorders. (nih.gov)
Specifically we will review the current state of science in gene based therapeutics including gene targeted approaches, vector design and delivery options, characteristics of suitable gene molecular targets and mechanisms amenable to gene based therapies. (nih.gov)
I'm really excited that we're hosting this workshop in an era where gene targeted therapeutics have begun to be applied in the nervous system and have already achieved FDA approval even in a couple of cases, and we need to think about approaches that will be appropriate to treat the disorders that NIMH is responsible for. (nih.gov)

Important methods of immunotherapy in cancer involve the use of cytokines, monoclonal antibodies, cancer vaccines and immunogene therapy. (researchandmarkets.com)
Dr. Fauci reviewed the types of HIV vaccine candidates that have been developed and tested, including viral subunit vaccines and vector-based vaccines containing HIV genes. (nih.gov)
1.Vero cell line is the most commonly used cell line for the production of viral vectors and vaccines. (ubigene.us)
This invention claims nine major salivary proteins from the sand fly vector of Leishmania major , Phlebotomus papatasi , nucleic acids encoding the proteins, vaccines comprising the proteins and/or nucleic acids, and methods of producing an immune response to prevent Leshmaniasis . (nih.gov)
mRNA vaccines have tremendous potential to fight against cancer and viral diseases due to superiorities in safety, efficacy and industrial production. (biomedcentral.com)
The article will discuss the molecular biology of mRNA vaccines and underlying anti-virus and anti-tumor mechanisms, with an introduction of their immunological phenomena, delivery strategies, their importance on Corona Virus Disease 2019 (COVID-19) and related clinical trials against cancer and viral diseases. (biomedcentral.com)
Multiple virus-oriented vaccines are currently used for routine vaccination, which gains significant progress in preventing and treating viral diseases. (biomedcentral.com)
Although much progress towards developing vaccines has been achieved, there still exist viral pathogens escaping the adaptive immune responses [ 5 ]. (biomedcentral.com)
These advantages enable mRNA-based vaccines a priority in the treatment of tumors and viral diseases. (biomedcentral.com)

Mutations of genes related to growth control, apoptosis, invasion and metastasis form the molecular genetic basis of malignant transformation and tumor progression[ 3 ]. (wjgnet.com)
However, one of the major obstacles in translating these basic science discoveries into safe cell based therapies for patients is the risk of acquiring mutations from viral and DNA vectors that are used to manipulate pluripotent cells into the specific cell types required to treat human diseases. (ca.gov)
[3] Mutations of FSHD cause inadequate DUX4 repression by unpacking the DNA around DUX4 , making it accessible to be copied into messenger RNA (mRNA). (mdwiki.org)

The early steps of HIV-1 infection involve numerous interactions between various viral proteins and host immune cells. (springeropen.com)
The LEAPER 2.0 technology utilizes the endogenous ADAR proteins present in cells to achieve proficient and precise RNA editing. (biomedcentral.com)
RNA-Seq), and constructing and imaging fluorescent proteins. (cshlpress.com)
Based on the level of sequence complementarity, miRNAs lead to the degradation of target mRNAs or the suppression of mRNA translation, thereby inhibiting the synthesis of proteins and achieving the regulation of genes. (biomedcentral.com)

In theory, the vectors have the potential to recombine with endogenous host or viral sequences to produce replication-competent virus. (the-scientist.com)
Although combinatorial antiretroviral therapy (cART) is capable of potently suppressing HIV-replication as well as delaying the onset of AIDS, the phenomenon of viral mutagenesis enables viral escape from these drugs [ 2 ]. (springeropen.com)
It is noted that very few direct recognition mechanisms of virus infections have been identified in Drosophila and that the activation of immune pathways may be accomplished indirectly through cell damage incurred by viral replication. (preprints.org)
Furthermore, the miR-3470b mimic transfection significantly contributed to increase the BEFV N mRNA, G protein level and viral titer, respectively, whereas the miR-3470b inhibitor had the opposite effect on BEFV replication. (biomedcentral.com)
Moreover, the overexpression of MAVS or silencing of miR-3470b by its inhibitors suppressed BEFV replication, and knockdown of MAVS by small interfering RNA also promoted the replication of BEFV. (biomedcentral.com)
Our findings is the first to reveal that miR-3470b as a novel host factor regulates BEFV replication via directly targeting the MAVS gene in BHK-21 cells and may provide a potential strategy for developing effective antiviral therapy. (biomedcentral.com)
[ 29 ] Inhibition of SAH indirectly inhibits transmethylation reactions required for viral replication. (medscape.com)
As Tat accumulates above the threshold, it leads to the transition from latent state of HIV-1 to active replication in lymphocytes when the protein interacts with the Tat-responsive element (TAR) located in the long terminal repeat (LTR) promoter in the viral DNA [ 9 - 11 ]. (biomedcentral.com)

Gene therapy is a promising molecular alternative in the treatment of gastric cancer, including the replacement of defective tumor suppressor genes, the inactivation of oncogenes, the introduction of suicide genes, genetic immunotherapy, anti-angiogenetic gene therapy, and virotherapy. (wjgnet.com)
However, little is known about how tumor cells co-opt cellular processes to express, from rearranged DNA, an in-frame fusion transcript encoding a chimeric functional protein and so we are using functional genetic approaches to identify genes required for the expression or activity of fusion oncoproteins. (nih.gov)
Mingozzi, F. & High, K. A. Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges. (nature.com)
We introduce the spectrum of genetic tools, delineate viral and nonviral CRISPR delivery strategies and describe a series of applications, ranging from gene editing and cancer modeling to chromosome engineering or CRISPR multiplexing and its spatio-temporal control. (nature.com)
Notably, patisiran and givosiran have received approval for the treatment of rare genetic disorders, such as hereditary Transthyretin-Mediated Amyloidosis (hATTR) and Acute Hepatic Porphyria (AHP), respectively. (scitechnol.com)
Gene technology: basic and applied molecular genetic methods. (lu.se)
Huntington's disease (HD) has become a target of the first clinical trials for gene therapy among movement disorders with a genetic origin. (e-jmd.org)
There is also SV40 promoter/enhancer in Moderna and Pfizer which is small segment of genetic code which initiates transcription from DNA to RNA and also amplifies the amount of RNA which is produced. (biblaridion.info)
FSHD is caused by a genetic mutation leading to deregulation of the DUX4 gene . (mdwiki.org)
The DUX4 gene was discovered in 1999, found to be expressed and toxic in 2007, and in 2010 the genetic mechanism causing its expression was elucidated. (mdwiki.org)

These include antiangiogenic agents, immunotherapy, bacterial agents, viral oncolysis, targeting of cyclic-dependent kinases and tyrosine kinase receptors, antisense approaches, gene therapy and combination of various methods. (researchandmarkets.com)
Traditional treatment approaches have been limited to managing symptoms, leaving a significant unmet medical need for targeted and curative therapies. (scitechnol.com)
Current approaches to manipulate stems cells into specific cell types or lineages requires exposure of stem cells to DNA vectors can result in integration of the DNA element into the chromosome of the stem cell and thereby potentially induce a non-desirable effect, including malignant mutation. (ca.gov)
So far, the list of FDA-approved gene therapy products is quite short, showing that the approaches to gene therapy research are experimental. (e-jmd.org)
Most approaches are currently targeted at the manifest HD stage, when clinical outcomes are used to evaluate the effectiveness of therapy. (biorxiv.org)
Approaches that use RNA marker dependent (conditional) activation of OGT agents may improve specificity but require additional validation in cell culture and in vivo . (thno.org)

Christoph, T. (2020) RNA Interference Mediated Silencing of Kv7.2 in Rat Dorsal Root Ganglion Neurons Abolishes the Antinociceptive Effect of a Selective Channel Opener. (tu.berlin)
Wells KL , Miller CN, Gschwind AR, Wei W, Phipps JD, Anderson MS and Steinmetz LM, 2020, Combined transient ablation and single-cell RNA- sequencing reveals the development of medullary thymic epithelial cells . (stanford.edu)
Ho EK, Tsai AE, Stearns T., 2020, Transient primary cilia mediate robust Hedgehog pathway-dependent cell cycle control, Current Biology , 30(14):2829-2835.e5. (stanford.edu)

19. Dominant-negative inhibition of Ets 1 suppresses tumor growth, invasion and migration in rat C6 glioma cells and reveals differentially expressed Ets 1 target genes. (nih.gov)
Generally, microRNAs have emerged as master regulators that typically bind to complementary sequences in the 3′ untranslated region (UTR) of multiple mRNA targets and modulate gene expression either via the translational inhibition or degradation of their target messenger RNAs (mRNAs) in a sequence-specific manner [ 6 , 7 , 8 ]. (biomedcentral.com)
The n-butanol fraction showed a dose-dependent inhibition against HIV-1 NL4.3 infection of the peripheral blood lymphocytes and against HIV-1 BaL (R-5-tropic) as well as two different primary viral isolates of HIV-1 infection of TZM-bl cells. (biomedcentral.com)
The results presented here show a potential anti-HIV-1 activity of A. catechu mediated by the inhibition of the functions of the viral protein and Tat. (biomedcentral.com)

The therapeutic outcome of conventional therapies is inefficient. (wjgnet.com)
The characterization of dysregulated genes like the tumor suppressor p53, which are critical for carcinogenesis, and a better understanding of the molecular basis for tumor-host interaction led to significant progress in the development of new therapeutic agents. (wjgnet.com)
More than 15 years ago, gene therapy emerged as a new therapeutic approach and has meanwhile become an important strategy in cancer treatment. (wjgnet.com)
Cancer is by far the most frequent of all indications addressed by gene therapy (60% of all clinical trials[ 3 ]), underlining the expectations raised by this new therapeutic option. (wjgnet.com)
14. Dominant-negative mutant of c-Jun gene transfer: a novel therapeutic strategy for colorectal cancer. (nih.gov)
This virus type presents several advantages over alternate vectors for therapeutic gene delivery. (nih.gov)
Therefore, we discuss some representative advances related to the application of viral- and nonviral-mediated miRNA delivery systems and provide a new perspective on the future of miRNA-based therapeutic strategies. (biomedcentral.com)
Oligonucleotide gene therapy (OGT) agents (e. g. antisense, deoxyribozymes, siRNA and CRISPR/Cas) are promising therapeutic tools. (thno.org)
CRISPR/Cas9 system can cause hundreds of non-intended dsDNA breaks due to low specificity of the guide RNA, which can limit therapeutic applications of CRISPR/Cas9 by ex-vivo formats. (thno.org)

Importantly, PTD-mediated delivery of peptide and protein cargo has now been tested in over 1,000 patients in multiple phase I and II clinical trials for heart disease, pain, and cancer. (ca.gov)
PMID- 19410251 TI - Association of C-reactive protein (CRP) gene allelic variants with serum CRP levels and hypertension in Turkish adults. (nih.gov)
In guinea pigs the Ad-CAGoptZGP and DEF201 combination therapy elicited full protection when treated 30 minutes post-exposure and were a superior treatment to Ad-CAGoptZGP supplemented with recombinant IFN-α protein. (omicsonline.org)
then, this RNA will go on to be translated into a protein that has a certain function in cells. (e-jmd.org)
The CAG repeats present in the gene will result in the translated protein Huntingtin (Htt) containing repeated stretches of glutamine that make it prone to misfolding and eventually cause the aggregation of the protein [ 2 , 3 ] that then loses its original function. (e-jmd.org)
Given the important role of ApoE in viral infections, we employed as well-studied model the hepatitis C virus (HCV) and found that the viral replicase component nonstructural protein 5A (NS5A) is enriched in ApoE-containing ILVs. (bvsalud.org)
Additionally, the target association between miR-3470b and mitochondrial antiviral signaling protein (MAVS) was predicted by target gene prediction tools and further validated using a dual-luciferase reporter assay, and the expression of MAVS mRNA and protein levels was negatively associated with miR-3470b levels. (biomedcentral.com)
This gene encodes a multi-domain protein involved in signaling and organization of cell junctions during embryogenesis. (cancerindex.org)
What does this gene/protein do? (cancerindex.org)
What pathways are this gene/protein implicaed in? (cancerindex.org)
Of the 1,785 seed genes analyzed, 13 prognostic feature genes, including collagen type XXVIII α1 chain (COL28A1), PDS5 cohesin‑associated factor A (PDS5A), zinc‑finger DHHC‑type containing 2 (ZDHHC2), zinc‑finger protein 24 (ZNF24), myosin VA (MYO5A) and myeloid/lymphoid or mixed‑lineage leukemia translocated to 4 (MLLT4), were identified. (cancerindex.org)
Huntington's disease (HD) is a dominantly inherited, fatal neurodegenerative disorder caused by a CAG expansion in the Huntingtin ( HTT ) gene, coding for pathologic mutant HTT protein (mHTT). (biorxiv.org)
[8] DUX4 protein is a modulator of hundreds of other genes, many of which are involved in muscle function. (mdwiki.org)
This small RNA can suppress the expression level of nuclear protein LIN-14 and thus regulates the development of nematodes [ 10 ]. (biomedcentral.com)
The late events of the viral life cycle include the processes of HIV-1 mRNA synthesis, protein expression and virus maturation. (biomedcentral.com)

Four patients have been treated with autologous T cells transfected with an antisense code directed against the HIV envelope RNA, says VIRxSYS founder Boro Dropulic. (the-scientist.com)
With technical advancements, gene transfer has been tried for several neurogenetic disorders, and now, gene therapy such as antisense oligonucleotides (ASOs) for spinal muscular atrophy has already been Food and Drug Administration (FDA) approved for clinical use. (e-jmd.org)
Notably, siRNA suppress non-targeted genes due to (1) the partial complementarity to mRNA 3'-untranslated regions (3'-UTR), and (2) the antisense activity of the sense strand. (thno.org)
The OGT agents, subjects of this review, are antisense oligonucleotides (ASO agents), small interfering RNA (siRNA), ribozymes (Rz), deoxyribozymes (Dz) and CRISPR/Cas. (thno.org)

Improved molecular biological techniques and a better understanding of gastric carcinogenesis have allowed us to validate a variety of genes as molecular targets for gene therapy. (wjgnet.com)
To identify radioresistant genes will provide novel targets for combined treatments and prognostic markers. (biomedcentral.com)
November 2012 - Researchers from NCATS designed a novel drug discovery method that uses two co-expressed reporter genes rather than one to increase the odds of identifying candidate compounds with true activity against biological or disease targets. (nih.gov)
Evidence that simple over-expression of any of the Runx gene family members can drive oncogenesis emerged first from mouse models, where it was shown that all three genes can act as targets for murine leukemia virus (MLV) insertional mutagenesis and transcriptional activation in lymphoma. (oncotarget.com)
Highly active antiretroviral therapy (HAART) has led to a dramatic increase in the longevity and the quality of life for people infected with HIV-1 [ 1 ], but due to the emergence of drug resistant virus [ 2 ], there is a continuous need to develop new anti-HIV-1 agents with novel targets and mechanisms of action. (biomedcentral.com)
Oligonucleotide-based gene therapy (OGT) is a variation of gene therapy that uses short synthetic DNA, RNA or their chemical analogs to hybridize to specific RNA or DNA targets followed by their inactivation. (thno.org)

Advanced topics include single-neuron isolation and analysis, in vivo gene delivery and imaging, optogenetics, RNA interference, transgenic technologies, high-throughput analysis of gene expression (e.g. (cshlpress.com)
Transcriptomes were evaluated using high-throughput RNA sequencing (RNA-seq) and real-time PCR (RT-PCR). (immunologiya-journal.ru)

In-depth characterization of antibody responses to viral infections in humans. (utmb.edu)

The endogenous adenosine deaminases acting on RNA (ADAR) have been harnessed to facilitate precise adenosine-to-inosine editing on RNAs. (biomedcentral.com)
MicroRNAs (miRNAs) are a class of endogenous and short noncoding RNAs (approximately 22 nucleotides in length) processed from endogenous transcripts. (biomedcentral.com)
After medical school he did a post-doctoral angiogenesis research fellowship at the Harvard Medical School with Dr. Judah Folkman, Anthony Adamis (Senior VP Global head of Ophthalmology/Immunology Roche), Joan Miller (Chair Harvard Medical School Ophthalmology), Patricia D'Amore ( Director of SERI), Robert D'Amato( Inventor of Revilimid) During this fellowship he helped develop a xenograft model system to identify endogenous anti-angiogenic therapies. (truebluevision.ca)

Therefore, non-viral cationic vectors (NVCVs) have received considerable attention to not only address these issues but also for developing efficacious siRNA delivery vectors. (nih.gov)

They are enveloped, single-stranded, negative-sense RNA viruses, which cause severe hemorrhagic fever and are associated with highly lethal infections in humans. (omicsonline.org)
Its causative agent, bovine ephemeral fever virus (BEFV) is an enveloped, nonsegmented, single stranded negative sense RNA virus in the Ephemerovirus genus of the Rhabdoviridae family. (biomedcentral.com)

[ 81 ] Vector-mediated intrastriatal growth factor overexpression alone can stimulate survival and or recruitment of SVZ neuroblasts in the adjacent striatum. (medscape.com)
GeneMedi's AAV4 Vector System (AAV serotype 4 helper-free packaging plasmids system) is including AAV4 Rep-Cap plamid (AAV4-RC plasmid, or called AAV-RC1 plasmid), AAV helper plasmid and AAV expression vectors (overexpression or shRNA). (genemedi.net)
The chief aim of this study is to understand the relationship between overexpression of drug resistance associated genes and multidrug resistance in ovarian cancer. (aacrjournals.org)

Furthermore, there are multiple changes in gene expression that occur during aging that hinder neurogenesis. (medscape.com)
[ 87 ] In another study, viral vector-mediated expression of bFGF/BDNF in the hippocampus of an epileptic lesion model increased neurogenesis, reduced neuronal damage and ameliorated epileptogenesis. (medscape.com)
van Vliet AC, Lee J, van der Poel M, Mason MRJ, Noordermeer JN, Fradkin LG, Tannemaat MR, Malessy MJA, Verhaagen J , De Winter F. Coordinated changes in the expression of Wnt pathway genes following human and rat peripheral nerve injury. (neurotree.org)
Melief J, Orre M, Bossers K, van Eden CG, Schuurman KG, Mason MRJ, Verhaagen J , Hamann J, Huitinga I. Transcriptome analysis of normal-appearing white matter reveals cortisol- and disease-associated gene expression profiles in multiple sclerosis. (neurotree.org)
4. Suppression of tumor growth, invasion and angiogenesis of human gastric cancer by adenovirus-mediated expression of NK4. (nih.gov)
12. Adenovirus-mediated expression of dominant negative c-myb induces apoptosis in head and neck cancer cells and inhibits tumor growth in animal model. (nih.gov)
18. Suppression of growth of pancreatic cancer cell and expression of vascular endothelial growth factor by gene silencing with RNA interference. (nih.gov)
GeneMedi's AAV expression vectors have been inserted with differernt expression cassettes, containing kinds of verified protomters and reporters including GFP, zsgreen, RFP, mcherry and luciferase. (genemedi.net)
You can also achieve gene knock-out(KO) or gene editing using our Crispr-cas9-gRNA AAV expression vector. (genemedi.net)
Moreover, a multiplexed mechanism of shRNAs and miRNA is known to be effective in preventing viral escape due to mutation as the miRNA develops a general polycistronic platform for the expression of a large amount of shRNA-miRs. (springeropen.com)
Principles of gene expression. (lu.se)
Molecular mechanisms for regulation of gene expression at different levels: remodeling of chromatin, initiation of transcription, nuclear transport and signalling, and RNA interference. (lu.se)
Methods for analysis of gene expression: microarray, hybridisation, promoter analyses. (lu.se)
Principles for regulation of gene expression through intercellular signalling. (lu.se)
Strategies for gene therapy and production of medicines via genetically-modified organisms (expression vectors and viral vectors). (lu.se)
Northern analysis shows that PRP-4 is overexpressed in several paclitaxel-resistant cell lines and confirms that PRP-4 expression could be significantly repressed by PRP-4 lentiviral short hairpin RNA. (aacrjournals.org)
Htt shuttles into the nucleus, but it is usually active in the cytoplasm, and it plays a role in vesicle transport and can regulate gene expression. (e-jmd.org)
This invention provides for synthetic dsRNAs (20-25 nucleotides in length) and methods that can inhibit gene-specific expression in mammalian cells. (nih.gov)
In this study, we found a set of microRNAs induced by BEFV infection using small RNA deep sequencing, and further identified BEFV infection could significantly up-regulate the miR-3470b expression in Baby Hamster Syrian Kidney cells (BHK-21) after 24 h and 48 h post-infection (pi) compared to normal BHK-21 cells without BEFV infection. (biomedcentral.com)
Search the gene expression profiles from curated DataSets in the Gene Expression Omnibus (GEO) repository. (cancerindex.org)
The present study aimed to construct a gene expression classifier and to screen key genes associated with GBM prognosis. (cancerindex.org)
Transcriptome analysis of Runx1 -deleted cells revealed a gene signature associated with lymphoid proliferation, survival and differentiation, and included strong de-repression of recombination-activating ( Rag) genes, an observation that was mirrored in a panel of human acute leukemias where RUNX1 and RAG1,2 mRNA expression were negatively correlated. (oncotarget.com)
miRNAs, which exhibit tissue- and temporal- specific expression, are important negative regulatory RNAs that decrease the levels of other functional genes. (biomedcentral.com)
MicroRNAs (miRNAs) comprise a group of small non-coding RNAs 18~25 nucleotides (nt) in length that posttranscriptionally regulate gene expression via binding to the 3′-untranslated regions (3′-UTRs) of target gene mRNA [ 1 , 2 ]. (biomedcentral.com)
The progeny viruses expressed from the activated viral gene expression are assembled on and budded through the host cell membrane after being processed by the viral encoded enzyme protease [ 7 ]. (biomedcentral.com)
Based on expression of selected genes, it is assumed that combined stimulation of NOD- and Toll-like receptors results in synergistic enhancement of cellular response (the effect of agonist combination is greater than sum of effects of individual agonists). (immunologiya-journal.ru)
Typical features of synergistically inducible genes were low basal expression and high response to combined stimulation. (immunologiya-journal.ru)
Results of bioinformatic analysis point at the role of NF- κ B and AP-1 transcription factor families in the regulation of expression of synergistically inducible genes. (immunologiya-journal.ru)

Convener of the Vaccine and Viral Vectors III Workshop, 28th Annual Meeting of American Society for Virology, University of British Columbia, Vancouver, Canada. (utmb.edu)
An effective vaccine is a powerful "weapon" for human to win this "fierce battle", and determining the host factors necessary for infection is very important for understanding the pathogenesis of COVID-19, revealing the changes of host susceptibility and determining new host oriented therapy, which may be effective for current and future pandemic Coronavirus. (ubigene.us)
Non-viral diseases, including cancer, need more vaccine-related researches to foster a novel vaccine development platform. (biomedcentral.com)

Detailed and comprehensive analysis of virus vectors are required to ensure their efficacy and safety. (chi-peptalk.com)
The whitefly Bemisia tabaci (Hemiptera: Aleyrodidae) is without doubt one of the most essential virus vectors, transmitting greater than 4 hundred virus species, the bulk belonging to begomoviruses (Geminiviridae), with their ssDNA genomes. (lcabie.com)

MicroRNAs (miRNAs) are naturally occurring, small non-coding RNAs that mediate posttranscriptional regulation. (biomedcentral.com)

Innovative cancer therapies are based on current concepts of the molecular biology of cancer. (researchandmarkets.com)
Institute of Molecular Pathobiochemistry, Experimental Gene Therapy and Clinical Chemistry (IFMPEGKC), RWTH Aachen University Hospital, Pauwelsstr. (mdpi.com)

Fusion between the virus as well as cellular membranes will result in viral capsid translocation into the cytoplasm. (springeropen.com)

Anti-PD-1 antibody therapy potently enhances the eradication of established tumors by gene-modified T cells. (ac.ir)

Lefesvre, P., Attema, J. & van Bekkum, D. A comparison of efficacy and toxicity between electroporation and adenoviral gene transfer. (nature.com)
Fig. 4: Efficacy of viral and nonviral nucleic acid delivery methods for the pancreas and liver of mice. (nature.com)

[ 82 , 83 ] Adeno-associated virus (AAV)-mediated delivery of BDNF enhanced the recruitment of progenitor cells to the lesioned striatum and promoted neuronal differentiation. (medscape.com)
Cancer gene therapy is a sophisticated form of drug delivery for cancer. (researchandmarkets.com)
Profiles of 238 companies involved in developing innovative cancer therapies and methods of delivery are presented along with their 291 collaborations. (researchandmarkets.com)
Figure 4: Nanoneedles mediate in vivo delivery. (nature.com)
Nishikawa, M. & Huang, L. Nonviral vectors in the new millennium: Delivery barriers in gene transfer. (nature.com)
None of these clinical trials have reported any ill effects or consequences of PTD-mediated delivery. (ca.gov)
The administration methods include (1) ex vivo delivery with the help of viral vectors, nanoparticles, and electroporation, (2) nonspecific in vivo delivery using non-viral carriers including liposomes, dendrimers and aptamers, as well as (3) targeted delivery that uses antibodies, modified nanoparticles, nucleic acid aptamers, and tissue-specific serotypes of AAV. (springeropen.com)
In this study, we demonstrate that AAV-mediated delivery of circular ADAR-recruiting RNAs (arRNAs) achieves effective RNA editing in non-human primates at dosages suitable for therapy. (biomedcentral.com)
In this study, we also endeavored to optimize the LEAPER 2.0 system further to establish robust RNA editing in non-human primates and humanized mice through AAV delivery at clinically reasonable doses. (biomedcentral.com)
One promising form of non-viral gene delivery system is liposomes. (mdpi.com)
AAV also shows good serotype specificity to various cell types for targeted gene delivery. (nih.gov)

The VIRxSYS trial is unique, in that this five-patient, Phase I safety trial is the first in which a lentiviral vector has been used to modify cells that are subsequently returned to a human subject. (the-scientist.com)
Using lentiviral short hairpin RNA collections targeting 132 genes identified from transcriptional profiling of drug-resistant cancer cell lines, individual knockdown experiments were done in the presence of sublethal doses of paclitaxel. (aacrjournals.org)
Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer. (ac.ir)

12-16) He was the first to demonstrate that the RNA interference mechanism worked in vertebrate organisms. (truebluevision.ca)
Because of its gain-of-function mechanism and monogenic etiology, strategies to lower HTT are being actively investigated as disease-modifying therapies. (biorxiv.org)

It belongs to a three-membered family of mammalian gene products that bind a common DNA target sequence by virtue of the conserved Runt domain and share a common heterodimeric binding co-factor, CBFβ [ 2 , 3 ]. (oncotarget.com)

Co-chair of the workshop "Interference with Host Defense", 40th Annual Meeting of American Society for Virology (virtual), July 23, 2021. (utmb.edu)

Several types of small regulatory RNA are discussed in this present study, including microRNA (miRNA), small interfering RNA (siRNA), and short hairpin RNA (shRNA). (springeropen.com)
Establishment of adoptive cell therapy with tumor infiltrating lymphocytes for non-small cell lung cancer patients. (ac.ir)

[ 76 ] The classical paradigm for gene therapy is the replacement of defective genes (loss of function) with the corrected gene in critical cell populations. (medscape.com)

He collaborated with Drs. Jim Wilson (Inventor of Ex Vivo Gene Therapy) and Jean Bennett (Inventor of Gene therapy for LCA) on the development of regulatable adeno-associated gene therapy vectors to deliver anti-angiogenic therapies. (truebluevision.ca)

Gene therapy's heady days of introducing the gene for adenosine deaminase into immune cells to treat a life-threatening congenital defect, as was done in the first gene therapy trial in 1990, have given way to an atmosphere of caution. (the-scientist.com)
The results highlight the importance of the innate immune response in the prevention of ZEBOV pathogenesis and support further development of the Ad-CAGoptZGP with DEF201 treatment combination for post-exposure therapy against ZEBOV infection. (omicsonline.org)

Aihara, H. & Miyazaki, J-I. Gene transfer into muscle by electroporation in vivo . (nature.com)
Six different sgRNA were designed and cloned in PX-458 plasmid vector, and PBMCs were transfected using lipofectamine 2000 and electroporation. (ac.ir)

This Funding Opportunity Announcement (FOA) issued by the National Heart, Lung, and Blood Institute (NHLBI), National Institutes of Health (NIH), invites applications from organizations/institutions that propose the development of new and efficient non-viral vectors that can overcome the limitations of viral vectors for gene therapy clinical trials in heart, lung, and blood diseases. (nih.gov)
In this review paper, the authors attempt to cover the characteristics of HD itself while providing an overview of the gene transfer methods currently being researched, and will introduce an experimental trial with a preclinical model of HD followed by an update on the ongoing clinical trials for patients with HD. (e-jmd.org)

These vectors are derived from pathogens such as HIV and its kin, and so the field proceeds with utmost caution. (the-scientist.com)

Notice the sequence coverage is deeper on the part of the vector that is expressed and has R-Loops. (biblaridion.info)
Double-stranded RNA (dsRNA) has been shown to trigger sequence-specific gene silencing in a wide variety of organisms, including plant, nematode and invertebrate species. (nih.gov)

Targetted correction of a mutant HPRT gene in mouse embryonic stem cells. (nature.com)
Use of induced-pluripotent (iPS) cell, embryonic stem cell, and adult stem cell research opens the door to promising new medical cell therapy applications and discoveries. (ca.gov)

Ubigene exclusive KO Cell Line Bank, over 5000 KO cell lines, covering thousands of genes from 8 popular signaling pathways and nearly 100 diseases. (ubigene.us)
These genes performed well on sample classification and prognostic risk differentiation, and six pathways, including adherens junction, cyclic adenosine 3',5'‑monophosphate signaling and Ras signaling pathways, were enriched for these feature genes. (cancerindex.org)

He then switched his research focus to retinal disease by studying Verteporfrin (Photodynamic Therapy) and vascular endothelial growth factor (VEGF). (truebluevision.ca)
It was during this time that he helped develop anti-vascular endothelial growth factor (VEGF) therapies such as Avastin, Lucentis Macugen and Eylea treatments used today for macular degeneration and diabetic retinopathy. (truebluevision.ca)

Seed genes were identified by the 'survival' package in R and subjected to pathway enrichment analysis. (cancerindex.org)

In 2007, there were 2.5 million new HIV infections worldwide, and for every person started on antiretroviral drug therapy, three become infected. (nih.gov)
1989, Viral Infections of Humans. (justia.com)

Many plant viruses depend upon insect vectors for his or her transmission and dissemination. (lcabie.com)

Gene therapy and the combination of gene therapy with direct stem cell manipulation have recently experienced a resurgence of optimism. (medscape.com)
The complementarity of stem cell and gene therapies is illustrated in Figure 2B & 2C. (medscape.com)
Consequently, to clinically develop stem cell therapies, there is a great need to develop reagents and protocols that can gently alter stem biology, but do not result in either use of DNA vectors or stress the cells, both of which can lead to mutagenic events. (ca.gov)
His research focused on gene, cellular and stem cell therapies. (truebluevision.ca)

15. Adeno-associated virus-mediated antiangiogenic gene therapy with thrombospondin-1 type 1 repeats and endostatin. (nih.gov)

Despite their relatively low number, this group of genes is of great functional importance, because it includes genes of key pro-inflammatory cytokines. (immunologiya-journal.ru)

Anatomy17

Organisms35

Diseases6

Chemicals and Drugs83

Analytical, Diagnostic and Therapeutic Techniques and Equipment37

Phenomena and Processes77

Disciplines and Occupations1

Technology, Industry, Agriculture2

Information Science4

Health Care2

RNAi37

SiRNAs4

Molecules3

Transduction6

Adeno-associated viral1

Genome9

Therapeutics4

Vaccines9

Mutations3

Proteins4

Replication8

Genetic10

Approaches6

20203

Inhibition4

Therapeutic9

Protein15

Antisense4

Targets6

High throughput2

Characterization1

Endogenous3

SiRNA delivery1

Single-stranded2

Overexpression3

Expression28

Vaccine3

Virus vectors2

Posttranscriptional1

Molecular2

Capsid1

Tumors1

Efficacy2

Delivery11

Lentiviral3

Mechanism2

Mammalian1

20211

Small2

Replacement of defective1

Vivo gene1

Immune2

Electroporation2

Clinical trials2

Pathogens1

Sequence2

Embryonic2

Pathways2

Vascular endoth2

Pathway1

Infections2

Viruses1

Stem cell4

Antiangiogenic1

Functional1