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Article

Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa.

Molecular therapy : the journal of the American Society of Gene Therapy - 2 Aug 2017

Fischer M Dominik, McClements Michelle E, Martinez-Fernandez de la Camara Cristina, Bellingrath Julia-Sophia, Dauletbekov Daniyar, Ramsden Simon C, Hickey Doron G, Barnard Alun R, MacLaren Robert E

Abstract excerpt

X-linked retinitis pigmentosa (XLRP) is generally a severe form of retinitis pigmentosa, a neurodegenerative, blinding disorder of the retina. 70% of XLRP cases are due to mutations in the retina-specific isoform of the gene encoding retinitis pigmentosa GTPase regulator (RPGRORF15). Despite successful RPGRORF15 gene replacement with adeno-associated viral (AAV) vectors being established in a number of animal...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 28549772
DOI: 10.1016/j.ymthe.2017.05.005
PMCID: PMC5542800

Genes

RPGR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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