Realizing Retinal Regeneration: How can data-driven in vivo reprogramming be used to treat retinal and optic nerve degeneration?

Approximately 1 in 2,000 people worldwide are affected by inherited retinopathies but few treatment options are available for retinal degeneration. The FDA’s 2017 approval of LUXTURNA to treat inherited retinal degeneration caused by biallelic mutations in RPE65 has established viral vectors as a viable clinical therapy to monogenic retinal disease. Furthermore, advances in pluripotent stem cell techniques have enabled retinal pigment epithelium (RPE) to reach clinical trials as a cell therapy for treating age-related macular degeneration (AMD).

Now it is time to talk about in vivo reprogramming: a novel therapeutic approach that will circumvent the issues with subretinal transplantation of cell therapies and the challenge of targeting every monogenic condition with gene therapy.