by University of California, Irvine In vivo correction of an Rpe65 mutation by adenine base editor (ABE) prolongs the survival of cones in LCA mouse model. Credit: Nature Communications (2022) A new University of California, Irvine-led study indicates base editing may provide long-lasting retinal protection and prevent vision deterioration in patients with inherited retinal degeneration, specifically in...
by National Eye Institute L-ORD patient RPE cell (borders are white) showing mutant protein CTRP5 (red) trapped inside the cell and partially in autophagosomes (green). Credit: Ruchi Sharma and Davide Ortolan (NEI) Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye...
Highlights iPSC retina reconstructs outer nuclear layer in the end-stage retina Contacts between the host bipolar cells and graft photoreceptors were visualized rd1 mice became responsive to light after iPSC-retina transplantation RGC responses to light were recorded from host rd1 retina after transplantation Summary Recent success in functional recovery by photoreceptor precursor transplantation in dysfunctional retina has led...
