Retinal organoids mirror CRISPR-Cas9 gene editing efficiency observed <i>in vivo</i>.

ABSTRACT: Human retinal organoids are in vitro 3D structures that recapitulate key molecular and structural characteristics of the in vivo retina. They include all essential retinal cell types including photoreceptors, making them relevant models for preclinical development of gene therapies. A critical knowledge gap exists in understanding their utility for gene editing therapy optimization. We assessed the potential of retinal organoids for optimizing CRISPR-Cas9-mediated gene editing, focusing on the therapeutically relevant RHO gene implicated in autosomal dominant retinitis pigmentosa (adRP). Using retinal organoids, in vitro HEK293T cells, and two humanized mouse models carrying different RHO mutations, we compared editing efficiencies. We observed that retin

SUBMITTER: Pulman J 

PROVIDER: S-EPMC12686912 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature