Results 31 to 40 of about 36,553 (255)
Scientific Reports, 2021 Available therapeutics for autoimmune disorders focused on mitigating symptoms, rather than treating the cause of the disorder. A novel approach using adeno-associated virus (AAV) could restore tolerance to the autoimmune targets and provide a permanent ...
Matthew Wade +3 more
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Adeno-associated virus integration: virus versus vector [PDF]
Gene Therapy, 2008 Although a large percentage of the world population is seropositive for exposure to various strains of adeno-associated virus (AAV), a human parvovirus, AAV has never been identified as an etiologic agent of human disease. Most likely contributing to the pronounced lack of pathogenicity is the fact that AAV is a naturally defective virus that requires ...
openaire +2 more sources
Adeno-associated virus vector integration junctions [PDF]
Journal of Virology, 1997 Vectors derived from adeno-associated virus (AAV) have the potential to stably transduce mammalian cells by integrating into host chromosomes. Despite active research on the use of AAV vectors for gene therapy, the structure of integrated vector proviruses has not previously been analyzed at the DNA sequence level.
E A, Rutledge, D W, Russell
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Adeno‐associated virus vectors for gene therapy—focusing on melanoma
Interdisciplinary MedicineAdeno‐Associated Virus (AAV) vectors have been found to have great potential in the field of gene therapy due to their unique properties. These nonpathogenic vectors exhibit high tissue specificity, low immunogenicity, and sustained gene expression ...
Xingyue Wang +6 more
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Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Onasemnogene abeparvovec (OA) is an AAV9‐based gene therapy for spinal muscular atrophy type I (SMA I). Real‐world outcomes show increased response variability compared to clinical trials, and follow‐up data beyond 12–18 months are limited.
Marika Pane +43 more
wiley +1 more source
BMC Biotechnology, 2007 Background Adjustable gene expression is crucial in a number of applications such as de- or transdifferentiation of cell phenotypes, tissue engineering, various production processes as well as gene-therapy initiatives.
Fussenegger Martin +2 more
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Frontiers in Cell and Developmental Biology, 2022 Direct reprogramming of retinal Müller glia is a promising avenue for replacing photoreceptors and retinal ganglion cells lost to retinal dystrophies. However, questions have recently been raised about the accuracy of studies claiming efficient glia-to ...
Nguyet Le +8 more
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Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Friedreich Ataxia (FRDA) is a neurodegenerative disorder of children and young adults associated with cardiomyopathy and other systemic complications. We report a 10‐year‐old girl who presented simultaneously with Acute Myelogenous Leukemia and FRDA who was successfully treated for her leukemia with allogeneic hematopoietic stem cell ...
Alexandra Gitman +5 more
wiley +1 more source
Scalable Production of AAV Vectors in Orbitally Shaken HEK293 Cells
Molecular Therapy: Methods & Clinical Development, 2019 Adeno-associated virus (AAV) vectors are currently among the most commonly applied for in vivo gene therapy approaches. The evaluation of vectors during clinical development requires the production of considerable amounts of highly pure and potent ...
Daniel Blessing +7 more
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Molecular Therapy: Methods & Clinical Development, 2020 Gene therapy with adeno-associated virus (AAV)-based vectors shows great promise for the gene therapeutic treatment of a broad array of diseases. In fact, the treatment of genetic diseases with AAV vectors is currently the only in vivo gene therapy ...
Alejandro Orlowski +5 more
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