Retinal transduction profiles by high-capacity viral vectors
نویسندگان
چکیده
منابع مشابه
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors.
Adeno-associated virus (AAV) vectors are effective gene delivery vehicles mediating long-lasting transgene expression. Data from a clinical trial of AAV2-mediated hepatic transfer of the Factor IX gene (F9) into hemophilia B subjects suggests that CTL responses against AAV capsid can eliminate transduced hepatocytes and prevent long-term F9 expression. However, the capacity of hepatocytes to pr...
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The development of fetal ocular gene transfer may be useful as a therapeutic tool for the prevention of retinal genetic disorders with congenital or early clinical manifestations. In this study we explored the neural progenitor transduction patterns of adeno-associated virus (AAV) vectors following delivery to the developing retina. Recombinant vectors with the same genome carrying the enhanced...
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BACKGROUND For both in vitro and in vivo gene transfer applications, recombinant viral vectors have almost always been used free in solution. Some site-specificity of the delivery of viral vectors can be achieved by applying a solution containing viral particles specifically to the site of interest. However, such site-specificity is seriously limited since viral vectors can diffuse freely in so...
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ژورنال
عنوان ژورنال: Gene Therapy
سال: 2014
ISSN: 0969-7128,1476-5462
DOI: 10.1038/gt.2014.57