Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial
Jean Bennett, Jennifer A. Wellman, Kathleen A. Marshall, Sarah McCague, Manzar Ashtari, Julie DiStefano‐Pappas, Okan Umit Elci, Daniel C. Chung, Junwei Sun, John Fraser Wright, Dominique Cross, Puya Aravand, Laura L. Cyckowski, Jeannette L. Bennicelli, Federico Mingozzi, Alberto Auricchio, Eric A. Pierce, Jason Ruggiero, Bart Peter Leroy, Francesca Simonelli, Katherine A. High, Albert M. Maguire
The abstract is not reproduced here because its license does not permit redistribution. Read it at the original source.
Record
- Archive ID
- doi:10.1016/s0140-6736(16)30371-3
- Published
- Jul 2, 2016
- Venue
- The Lancet
- DOI
- 10.1016/s0140-6736(16)30371-3
- PubMed
- 27375040
- Categories
- Retinal Development and Disorders
- Provenance
- openalex