Botaretigene sparoparvovec (AAV5-RPGR) is an experimental gene therapy for X-linked retinitis pigmentosa developed by Janssen Pharmaceuticals.[1][2]
References
edit- ^ 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 (2022-06-01). "AAV5-RPGR (botaretigene sparoparvovec) gene therapy for X-linked retinitis pigmentosa (XLRP) demonstrates localized improvements in static perimetry". Investigative Ophthalmology & Visual Science. 63 (7). The Association for Research in Vision and Ophthalmology: 3846. ISSN 1552-5783. Retrieved 2023-12-05.
- ^ "Humoral immune response to AAV5-RPGR (botaretigene sparoparvovec) gene therapy in RPGR-associated X-linked retinitis pigmentosa". Investigative Ophthalmology & Visual Science.