Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models.
使用双AAV内含子载体的视网膜基因疗法治疗Stargardt病在大动物模型中安全有效
期刊:Science Advances
影响因子:12.5
doi:10.1126/sciadv.adt9354
Ferla Rita, Pugni Eugenio, Lupo Mariangela, Tiberi Paola, Fioretto Federica, Perota Andrea, Duchi Roberto, Lagutina Irina, Gesualdo Carlo, Rossi Settimio, Ventrella Domenico, Elmi Alberto, McClinton Benjamin, Toomes Carmel, Xu Tongzhou, Molday Robert S, Surace Enrico M, Simonelli Francesca, Bacci Maria L, Galli Cesare, Memon Muhammad A, Shams Naveed, Auricchio Alberto, Trapani Ivana
