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

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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Bibliographic Details
Main Authors: 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
Format: Online
Language:English
Published: Oxford University Press 2015
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4634381/

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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4634381/

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