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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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書誌詳細
主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: 2014
主題:
オンライン・アクセス: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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