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Article

A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration.

Human molecular genetics - 15 Jul 2015

Wu Zhijian, Hiriyanna Suja, Qian Haohua, Mookherjee Suddhasil, Campos Maria M, Gao Chun, Fariss Robert, Sieving Paul A, Li Tiansen, Colosi Peter, Swaroop Anand

Abstract excerpt

Mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene account for >70% of X-linked retinitis pigmentosa (XLRP) and 15-20% of all inherited retinal degeneration. Gene replacement therapy for RPGR-XLRP was hampered by the relatively slow disease progression in mouse models and by difficulties in cloning the full-length RPGR-ORF15 cDNA that includes a purine-rich 3'-coding region; however, its...

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Identifiers

PMID: 25877300
DOI: 10.1093/hmg/ddv134
PMCID: PMC4476444

Genes

RPGR

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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