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Genome-Editing Strategies for Treating Human Retinal Degenerations

30 Sept 2020

Abstract excerpt

Inherited retinal degenerations (IRDs) are a leading cause of blindness. Although gene-supplementation therapies have been developed, they are only available for a small proportion of recessive IRD mutations. In contrast, genome editing using clustered-regularly interspaced short palindromic repeats (CRISPR) CRISPR-associated (Cas) systems could provide alternative therapeutic avenues for treating a wide range of...

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PMID: 32993386
DOI: 10.1089/hum.2020.231

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