From Dogs to Humans: Gene Therapy for Inherited Eye Disease Advances to Clinical Trials
A gene therapy that has been successful in treating an inherited, blinding eye disease in dogs is now ready for clinical trials in humans with the rare condition retinitis pigmentosa. The therapy introduces a normal copy of the CNGB1 gene, halting vision loss and potentially benefiting around 2 million people affected worldwide. Retinitis pigmentosa affects approximately 2 million people worldwide, with 100,000 cases in the US alone. The therapy has been successfully trialed in dogs and works by rescuing normal function in rod cells, preserving cone function, and preserving retinal structure by stopping photoreceptor degeneration.