Subretinal Gene Therapy for X-Linked Retinoschisis
Published June 10, 2026
N Engl J Med 2026;394:2223-2234
DOI: 10.1056/NEJMoa2515953
Abstract
Background
X-linked retinoschisis is a recessive disease characterized by progressive macular degeneration and vision loss due to pathogenic variation in RS1.
Methods
We administered a single subretinal injection of an AAV8 vector containing human RS1 compl...
