Botaretigene sparoparvovec explained

Botaretigene sparoparvovec (AAV5-RPGR) is an experimental gene therapy for X-linked retinitis pigmentosa developed by Janssen Pharmaceuticals.^{[1] [2]}

Notes and References

1. Michaelides . Michel . Xu . Jialin . Wang . Dai . Wong . Peggy . Fung . Albert . Forbes . Alexandra . Naylor . Stuart . Zeldin . Robert . Parker . Maria A . Weleber . Richard . Guimaraes . Thales Antonio Cabral de . Besirli . Cagri . Yang . Yesa . Bainbridge . James . AAV5-RPGR (botaretigene sparoparvovec) gene therapy for X-linked retinitis pigmentosa (XLRP) demonstrates localized improvements in static perimetry . Investigative Ophthalmology & Visual Science . The Association for Research in Vision and Ophthalmology . 63 . 7 . 2022-06-01 . 1552-5783 . 3846 . 2023-12-05.
2. Humoral immune response to AAV5-RPGR (botaretigene sparoparvovec) gene therapy in RPGR-associated X-linked retinitis pigmentosa . Investigative Ophthalmology & Visual Science .