Generation of Optogenetically Modified Adenovirus Vector for Spatiotemporally Controllable Gene Therapy

ACS Chemical BiologyDOI: 10.1021/acschembio.7b01058
Source: ACS Chemical Biology - Category: Chemistry Authors: Source Type: research

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Amyotrophic Lateral Sclerosis (ALS) is an incurable motor neuron (MN) disorder, characterized by degeneration of MNs leading to progressive paralysis and death within two to five years after diagnosis. Gene therapy is emerging as promising treatment option for patients affected by familial forms of ALS (fALS), representing 10% of all ALS cases. Taking advantage of viral vectors derived from Adeno-Associated Virus serotype 10 (AAV10) and the small nuclear RNA U7, we developed a gene therapy for SOD1-linked ALS (20% of fALS).
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
Gene Therapy, Published online: 30 September 2019; doi:10.1038/s41434-019-0104-5Superior human hepatocyte transduction with adeno-associated virus vector serotype 7
Source: Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
ConclusionThe findings of this study reveal that rAAV1-based vectors have high application potential for endothelial-targeted neurologic disease research or gene-based therapies.
Source: Journal of Neuroscience Methods - Category: Neuroscience Source Type: research
Human Gene Therapy, Ahead of Print.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Human Gene Therapy,Volume 30, Issue 10, Page 1297-1305, October 2019.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Human Gene Therapy,Volume 30, Issue 10, Page 1284-1296, October 2019.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Expands AAV gene therapy platform from inside the cell for genetic disorders RESEARCH TRIANGLE PARK, N.C., Sept. 17, 2019 -- (Healthcare Sales &Marketing Network) -- Asklepios BioPharmaceutical, Inc. (AskBio), a fully integrated Adeno-Associated Virus... Biopharmaceuticals, Drug Delivery, Acquisitions Asklepios BioPharmaceutical, AskBio, RoverMed BioSciences
Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news
Human Gene Therapy, Ahead of Print.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Patients with T-cell immunodeficiencies are generally treated with allogeneic hematopoietic stem cell transplantation, but alternatives are needed for patients without matched donors. An innovative intrathymic gene therapy approach that directly targets the thymus might improve outcomes.
Source: Journal of Allergy and Clinical Immunology - Category: Allergy & Immunology Authors: Source Type: research
Pioneering pulmonologists Dr. Steven Albelda and Dr. Daniel Sterman have worked for more than 20 years on developing gene therapy to effectively combat pleural mesothelioma cancer. The payoff may have finally arrived. Albelda and Sterman’s long-awaited, phase III clinical trial will open this month to evaluate the efficacy of TR002, a novel gene therapy drug, when used in combination with celecoxib and gemcitabine. TR002, a form of immunotherapy, is a genetically engineered adenovirus that triggers the anti-tumor effects of interferon, a naturally occurring protein that destroys cancer cells. “The hope is, if i...
Source: Asbestos and Mesothelioma News - Category: Environmental Health Authors: Source Type: news
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