Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease
改进的双 AAV 载体减少了截短蛋白的表达,在 Stargardt 病小鼠模型的视网膜中是安全有效的
期刊:Human Molecular Genetics
影响因子:3.1
doi:10.1093/hmg/ddv386
Ivana Trapani, Elisabetta Toriello, Sonia de Simone, Pasqualina Colella, Carolina Iodice, Elena V Polishchuk, Andrea Sommella, Linda Colecchi, Settimio Rossi, Francesca Simonelli, Massimo Giunti, Maria L Bacci, Roman S Polishchuk, Alberto Auricchio