Gene therapy research highlights X-linked RP associated with RPGR
Reviewed by Michel Michaelides, MD Botaretigene sparoparvovec gene therapy (formerly AAV5-RPGR; MeiraGTx Holdings plc and Janssen Pharmaceuticals, Inc) for the treatment of a common and severe form of retinitis pigmentosa (RP), RPGR– associated X-linked RA, demonstrated improvements in retinal sensitivity and visual function compared to controls and had a manageable adverse event profile, according to …
Gene therapy research highlights X-linked RP associated with RPGR Read More »