Posted innews Ophthalmology
Optimized AAV5-RPGR Gene Therapy Rescues Photoreceptor Integrity and Function in X-Linked Retinitis Pigmentosa Mouse Model
An optimized AAV5 vector delivering a stabilized human RPGR ORF15 gene significantly restores photoreceptor structure and function in RPGR-deficient mice, demonstrating a promising gene therapy for X-linked retinitis pigmentosa.