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Research article

First published 2017-3

Novel Variants of RPGR in X-Linked Retinitis Pigmentosa Families and Genotype-Phenotype Correlation

Francesco Parmeggiani, Vanessa Barbaro, Angelo Migliorati [...]

European Journal of Ophthalmology

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Research article

First published 2018-12

Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice

Chunjuan Song, Thomas J Conlon, Wen-Tao Deng [...]

Human Gene Therapy Clinical Development

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Research article

First published 2019-08

Development of a Molecularly Stable Gene Therapy Vector for the Treatment of RPGR -Associated X-Linked Retinitis Pigmentosa

Joseph C. Giacalone, Jeaneen L. Andorf, Qihong Zhang [...]

Human Gene Therapy

Open access

Research article

First published 2022-08

Exon Skipping Through Chimeric Antisense U1 snRNAs to Correct Retinitis Pigmentosa GTPase-Regulator ( RPGR ) Splice Defect

Giuseppina Covello, Gehan H. Ibrahim, Niccolò Bacchi [...]

Nucleic Acid Therapeutics

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Research article

First published 2020-07

Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy

Chunjuan Song, Valérie L. Dufour, Artur V. Cideciyan [...]

Human Gene Therapy

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Research article

First published 2020-02

Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa

Valérie L. Dufour

Human Gene Therapy

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Research article

First published 2015-09

Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa

Wen-Tao Deng, Frank M. Dyka, Astra Dinculescu [...]

Human Gene Therapy

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Letter

First published 2019-3

Re: Song et al.,Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice. Hum Gene Ther Clin Dev 2018;29(4):188–197

Robert E. MacLaren, M. Dominik Fischer, Cristina Martinez-Fernandez De La Camara

Human Gene Therapy Clinical Development

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Reply

First published 2019-3

Response to MacLaren et al .,re: Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice. Hum Gene Ther Clin Dev 2018;29(4):188–197

Guo-Jie Ye, Chunjuan Song, Jeff Chulay [...]

Human Gene Therapy Clinical Development

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Review article

First published 2013-03

Zebrafish Model for the Genetic Basis of X-Linked Retinitis Pigmentosa

Rakesh Kotapati Raghupathy, Daphne L. McCulloch, Saeed Akhtar [...]

Zebrafish

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