Article

RHO1-2 meganuclease gene editing targets human P23H rhodopsin-induced retinitis pigmentosa to rejuvenate rods and maintain cones

2025-09-25

Abstract excerpt

<h4>ABSTRACT</h4> Autosomal dominant retinitis pigmentosa (adRP) is an inherited retinal dystrophy characterized by progressive vision loss and eventual blindness. The P23H mutation (proline to histidine substitution at codon 23) in the rhodopsin (RHO) gene represents the most common form of adRP in North Americans. Currently, there is no cure for P23H adRP. Genome editing targeting the mutant RHO allele, leaving...

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Literature Corpus work: 54399485-428b-5128-a510-d6b6b99a77a7
DOI: 10.1101/2025.09.25.678625

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