MeiraGTx announced encouraging news regarding their #genetherapy treatment for AIPL1-related retinal dystrophy, LCA4, a retinal dystrophy that leaves children with only the ability to distinguish light and dark at birth. 4 out of 4 young children with LCA4 benefited from unilateral subretinal administration in the FIH trial, with improved visual acuity, functional vision, and protection against progressive retinal degeneration.
?? Gene Therapy Restores Vision in Children with Genetic Blindness ??? A groundbreaking gene therapy has given four children with AIPL1 deficiency, a severe form of retinal dystrophy, life-changing improvements in vision. Developed by UCL Institute of Ophthalmology and Moorfields Eye Hospital, with support from MeiraGTx, this treatment offers new hope for genetic blindness. ?? Key Findings: ? Healthy copies of the AIPL1 gene were delivered into retinal cells, helping them function and survive. ? All four children saw significant vision improvements in the treated eye over three to four years. ? Published in The Lancet, the study highlights how early gene therapy can transform lives. With gene therapy for RPE65 deficiency already available through the NHS, this breakthrough could pave the way for wider treatments for genetic blindness. ?? Read more here: https://lnkd.in/dUathC98 #GeneTherapy #VisionLoss #ScientificResearch #Innovation #Ophthalmology
