Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice

Vectors derived from adeno-associated virus (AAV) are promising for human gene therapy, including treatment for retinal blindness. One major limitation of AAVs as vectors is that AAV cargo capacity has been considered to be restricted to 4.7 kb. Here we demonstrate that vectors with an AAV5 capsid (...

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Detalhes bibliográficos
Main Authors: Allocca, Mariacarmela, Doria, Monica, Petrillo, Marco, Colella, Pasqualina, Garcia-Hoyos, Maria, Gibbs, Daniel, Kim, So Ra, Maguire, Albert, Rex, Tonia S., Di Vicino, Umberto, Cutillo, Luisa, Sparrow, Janet R., Williams, David S., Bennett, Jean, Auricchio, Alberto
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2008
Assuntos:
Technical Advance
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2298836/
https://ncbi.nlm.nih.gov/pubmed/18414684
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI34316
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