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Article

Autosomal Dominant Retinitis Pigmentosa Due to Class B Rhodopsin Mutations: An Objective Outcome for Future Treatment Trials.

International journal of molecular sciences - 27 Oct 2019

Sumaroka Alexander, Cideciyan Artur V, Charng Jason, Wu Vivian, Powers Christian A, Iyer Bhavya S, Lisi Brianna, Swider Malgorzata, Jacobson Samuel G

Abstract excerpt

Gene therapy for adRP due to RHO mutations was recently shown to prevent photoreceptor death in a canine model of Class B disease. Among translational steps to be taken, one is to determine a method to detect efficacy in a human clinical trial. The relatively slow progression of adRP becomes a difficulty for clinical trials requiring an answer to whether there is slowed progression of degeneration in response to...

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Identifiers

PMID: 31717845
DOI: 10.3390/ijms20215344
PMCID: PMC6861901

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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