Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.

doi:10.1016/j.ymthe.2017.05.004

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Article

Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.

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

Beltran William A, Cideciyan Artur V, Boye Shannon E, Ye Guo-Jie, Iwabe Simone, Dufour Valerie L, Marinho Luis Felipe, Swider Malgorzata, Kosyk Mychajlo S, Sha Jin, Boye Sanford L, Peterson James J, Witherspoon C Douglas, Alexander John J, Ying Gui-Shuang, Shearman Mark S, Chulay Jeffrey D, Hauswirth William W, Gamlin Paul D, Jacobson Samuel G, Aguirre Gustavo D

Abstract excerpt

X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene is an early onset and severe cause of blindness. Successful proof-of-concept studies in a canine model have recently shown that development of a corrective gene therapy for RPGR-XLRP may now be an attainable goal. In preparation for a future clinical trial, we have here optimized the therapeutic AAV vector construct by showing that GRK1...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 28566226
DOI: 10.1016/j.ymthe.2017.05.004
PMCID: PMC5542804

Genes

RPGR
GRK1

Provenance

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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