Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa

Autor: MICHAELIDES, MICHEL, BESIRLI, CAGRI G., YANG, YESA, DE GUIMARAES, THALES A.C., WONG, SUI CHIEN, HUCKFELDT, RACHEL M., COMANDER, JASON I., SAHEL, JOSÉ-ALAIN, SHAH, SYED MAHMOOD, TEE, JAMES J.L., KUMARAN, NERUBAN, GEORGIADIS, ANASTASIOS, MINNICK, PANSY, ZELDIN, ROBERT, NAYLOR, STUART, XU, JIALIN, CLARK, MICHAEL, ANGLADE, EDDY, WONG, PEGGY, FLECK, PENNY R., FUNG, ALBERT, PELUSO, COLLEEN, KALITZEOS, ANGELOS, GEORGIOU, MICHALIS, RIPAMONTI, CATERINA, SMITH, ALEXANDER J., ALI, ROBIN R., FORBES, ALEXANDRIA, BAINBRIDGE, JAMES
Zdroj: In American Journal of Ophthalmology November 2024 267:122-134
Databáze: ScienceDirect
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