Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.

Maeder, Morgan L; Stefanidakis, Michael; Wilson, Christopher J; Baral, Reshica; Barrera, Luis Alberto; Bounoutas, George S; Bumcrot, David; Chao, Hoson; Ciulla, Dawn M; DaSilva, Jennifer A; Dass, Abhishek; Dhanapal, Vidya; Fennell, Tim J; Friedland, Ari E; Giannoukos, Georgia; Gloskowski, Sebastian W; Glucksmann, Alexandra; Gotta, Gregory M; Jayaram, Hariharan; Haskett, Scott J; Hopkins, Bei; Horng, Joy E; Joshi, Shivangi; Marco, Eugenio; Mepani, Rina; Reyon, Deepak; Ta, Terence; Tabbaa, Diana G; Samuelsson, Steven J; Shen, Shen; Skor, Maxwell N; Stetkiewicz, Pam; Wang, Tongyao; Yudkoff, Clifford; Myer, Vic E; Albright, Charles F; Jiang, Haiyan.
Nat Med; 25(2): 229-233, 2019 02.
Artigo em Inglês | MEDLINE | ID: mdl-30664785