newsAAV5gene therapy
Optimized AAV5-RPGR Gene Therapy Rescues Photoreceptor Integrity and Function in X-Linked Retinitis Pigmentosa Mouse Model
By MedXY|Jun 13, 2026
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.