西亚试剂：Readministration in Three Adults with Congenital

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

Jean Bennett, Manzar Ashtari, Jennifer Wellman, Kathleen A. Marshall,Laura L. Cyckowski,Daniel C. Chung, Sarah McCague, Eric A. Pierce, Yifeng Chen, Jeannette L. Bennicelli, Xiaosong Zhu, Gui-shuang Ying, Junwei Sun, J. Fraser Wright,Alberto Auricchio, Francesca Simonelli, Kenneth S. Shindler, Federico Mingozzi,Katherine A. High and Albert M. Maguire

Abstract Demonstration of safe and stable reversal of blindness after a single unilateral subretinal injection of a recombinant adeno-associated virus (AAV) carrying the RPE65 gene (AAV2-hRPE65v2) prompted us to determine whether it was possible to obtain additional benefit through a second administration of the AAV vector to the contralateral eye. Readministration of vector to the second eye was carried out in three adults with Leber congenital amaurosis due to mutations in the RPE65 gene 1.7 to 3.3 years after they had received their initial subretinal injection of AAV2-hRPE65v2. Results (through 6 months) including evaluations of immune response, retinal and visual function testing, and functional magnetic resonance imaging indicate that readministration is both safe and efficacious after previous exposure to AAV2-hRPE65v2.