AAV.IVT18-mediated RDH12 gene therapy restores retinal metabolism in Rdh12 knockout rats and patient-derived iPSC-retinal organoids
Biallelic variants in retinol dehydrogenase 12 ( RDH12 ) cause severe inherited retinal diseases (IRDs) with no available treatments. Given the potential risks of subretinal delivery in patients with macular atrophy, we evaluated intravitreal AAV-mediated RDH12 gene augmentation. A Rdh12 -knockout rat model and patient...
Yunyu Zhou, Yamei Li, Yanan Shi et al.
· Stem Cell Research & Therapy · 0 citations