Article

Retinal organoids mirror CRISPR/Cas9 gene editing efficiency observed in vivo

2024-12-26

Abstract excerpt

Human retinal organoids are in vitro 3D structures that recapitulate key molecular and structural characteristics of the in vivo retina. They include the presence of 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 optimization, particularly for spec...

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Literature Corpus work: 07849ac8-6168-5ec3-a1fe-7291c312b1d1
DOI: 10.1101/2024.12.26.630388

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