Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.

doi:10.1073/pnas.1118847109

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Article

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.

Proceedings of the National Academy of Sciences of the United States of America - 7 Feb 2012

Beltran William A, Cideciyan Artur V, Lewin Alfred S, Iwabe Simone, Khanna Hemant, Sumaroka Alexander, Chiodo Vince A, Fajardo Diego S, Román Alejandro J, Deng Wen-Tao, Swider Malgorzata, Alemán Tomas S, Boye Sanford L, Genini Sem, Swaroop Anand, Hauswirth William W, Jacobson Samuel G, Aguirre Gustavo D

Abstract excerpt

Hereditary retinal blindness is caused by mutations in genes expressed in photoreceptors or retinal pigment epithelium. Gene therapy in mouse and dog models of a primary retinal pigment epithelium disease has already been translated to human clinical trials with encouraging results. Treatment for common primary photoreceptor blindness, however, has not yet moved from proof of concept to the clinic. We evaluated...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Identifiers

PMID: 22308428
DOI: 10.1073/pnas.1118847109
PMCID: PMC3277562

Genes

RPGR
GRK1

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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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