AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression
نویسندگان
چکیده
منابع مشابه
High-level transgene expression by homologous recombination-mediated gene transfer
Gene transfer and expression in eukaryotes is often limited by a number of stably maintained gene copies and by epigenetic silencing effects. Silencing may be limited by the use of epigenetic regulatory sequences such as matrix attachment regions (MAR). Here, we show that successive transfections of MAR-containing vectors allow a synergistic increase of transgene expression. This finding is par...
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Nowadays, there is no effective treatment for many retinal disorders. Knowledge of the genetic basis of many severe ocular diseases may allow for alternative treatments by gene therapy. Non-viral gene complexes, such as lipo- and poly-plexes, can be delivered to the posterior segment, most often the target tissue, by intravitreal or subretinal injection. Since subretinal injections are very inv...
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Transgenic mice have provided invaluable information about gene function and regulation. However, because of marked differences between rodents and primates, some areas of human biology such as early embryonic development, aging, and maternal-fetal interactions would be best studied in a nonhuman primate model. Here, we report that gene transfer into rhesus monkey (Macaca mulatta) preimplantati...
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BACKGROUND AND PURPOSE Gene therapy may be a promising approach for treatment of brain ischemia, although protein synthesis is generally inhibited in ischemic conditions. Our goal in this study was to examine effects of brain ischemia on transgene expression of adenovirus-mediated gene transfer to ischemic brain. METHODS Brain ischemia was produced by photochemical occlusion of the distal mid...
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Gene therapy for cystic fibrosis using non-viral, plasmid-based formulations has been the subject of intensive research for over two decades but a clinically viable product has yet to materialise in large part due to inefficient transgene expression. Minicircle DNA give enhanced and more persistent transgene expression compared to plasmid DNA in a number of organ systems but has not been assess...
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ژورنال
عنوان ژورنال: Journal of Clinical Investigation
سال: 2019
ISSN: 0021-9738,1558-8238
DOI: 10.1172/jci129085