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Article

In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2016

Bakondi Benjamin, Lv Wenjian, Lu Bin, Jones Melissa K, Tsai Yuchun, Kim Kevin J, Levy Rachelle, Akhtar Aslam Abbasi, Breunig Joshua J, Svendsen Clive N, Wang Shaomei

Abstract excerpt

Reliable genome editing via Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9 may provide a means to correct inherited diseases in patients. As proof of principle, we show that CRISPR/Cas9 can be used in vivo to selectively ablate the rhodopsin gene carrying the dominant S334ter mutation (Rho(S334)) in rats that model severe autosomal dominant retinitis pigmentosa. A single subretinal...

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Source

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Identifiers

PMID: 26666451
DOI: 10.1038/mt.2015.220
PMCID: PMC4786918

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

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