Optic vesicle-like structures derived from human pluripotent stem cells facilitate a customized approach to retinal disease treatment
Genome-Wide CRISPR Screening Identifies Cellular Factors Controlling Nonviral Genome Editing Efficiency
Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
Human iPSC Modeling Reveals Mutation-Specific Responses to Gene Therapy in a Genotypically Diverse Dominant Maculopathy
In situ autofluorescence lifetime assay of a photoreceptor stimulus response in mouse retina and human retinal organoids
Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy
