# 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

Article · 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

- Archive id: doi:10.1016/s0140-6736(16)30371-3
- Published: 2016-07-02
- Venue: The Lancet
- DOI: https://doi.org/10.1016/s0140-6736(16)30371-3
- PMID: 27375040
- Citations: 461
- Source URL: https://doi.org/10.1016/s0140-6736(16)30371-3
- Provenance: openalex

_Abstract not reproduced: its license does not permit redistribution. See the source URL._

Topics: [Gene & Cell Therapy](https://space-kitty.com/topics/gene-cell-therapy)

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