Retinal transduction profiles by high-capacity viral vectors

Retinal gene therapy with adeno-associated viral (AAV) vectors is safe and effective in humans. However, the limited cargo capacity of AAV prevents their use for therapy of those inherited retinopathies (IRs) due to mutations in large (>5kb) genes. Viral vectors derived from Adenovirus (Ad), Lent...

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Main Authors: Puppo, Agostina, Cesi, Giulia, Marrocco, Elena, Piccolo, Pasquale, Jacca, Sarah, Shayakhmetov, Dmitry M., Parks, Robin J., Davidson, Beverly L., Colloca, Stefano, Brunetti-Pierri, Nicola, Ng, Philip, Donofrio, Gaetano, Auricchio, Alberto
Format: Artigo
Jezik:Inglês
Izdano: 2014
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Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC4193889/
https://ncbi.nlm.nih.gov/pubmed/24989814
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2014.57
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