Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease.
Trapani I, Toriello E, de Simone S, Colella P, Iodice C, Polishchuk EV, Sommella A, Colecchi L, Rossi S, Simonelli F, Giunti M, Bacci ML, Polishchuk RS, Auricchio A.
Trapani I, et al. Among authors: polishchuk ev, polishchuk rs.
Hum Mol Genet. 2015 Dec 1;24(23):6811-25. doi: 10.1093/hmg/ddv386. Epub 2015 Sep 29.
Hum Mol Genet. 2015.
PMID: 26420842
Free PMC article.