Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65

Por Georgiadis, A, Duran, Y, Ribeiro, J, Abelleira-Hervas, L, Robbie, S J, Sünkel-Laing, B, Fourali, S, Gonzalez-Cordero, A, Cristante, E, Michaelides, M, Bainbridge, J W B, Smith, A J, Ali, R R
Publicado no Gene Ther (2016)