Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

Stargardt disease (STGD1) due to mutations in the large ABCA4 gene is the most common inherited macular degeneration in humans. We have shown that dual adeno-associated viral (AAV) vectors effectively transfer ABCA4 to the retina of Abca4−/− mice. However, they express both lower levels of transgene...

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Detalles Bibliográficos
Autores principales: Trapani, Ivana, Toriello, Elisabetta, de Simone, Sonia, Colella, Pasqualina, Iodice, Carolina, Polishchuk, Elena V., Sommella, Andrea, Colecchi, Linda, Rossi, Settimio, Simonelli, Francesca, Giunti, Massimo, Bacci, Maria L., Polishchuk, Roman S., Auricchio, Alberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2015
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4634381/
https://www.ncbi.nlm.nih.gov/pubmed/26420842
http://dx.doi.org/10.1093/hmg/ddv386

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4634381/
https://www.ncbi.nlm.nih.gov/pubmed/26420842
http://dx.doi.org/10.1093/hmg/ddv386

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