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Article

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

Human gene therapy - 1 Jul 2020

Song Chunjuan, Dufour Valérie L, Cideciyan Artur V, Ye Guo-Jie, Swider Malgorzata, Newmark Judith A, Timmers Adrian M, Robinson Paulette M, Knop David R, Chulay Jeffrey D, Jacobson Samuel G, Aguirre Gustavo D, Beltran William A, Shearman Mark S

Abstract excerpt

Recombinant adeno-associated viral (rAAV) vector-mediated gene therapy is being developed to treat X-linked retinitis pigmentosa (XLRP) in patients with mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. In preparation for a clinical gene therapy trial, we conducted dose range finding (DRF) studies with an AAV2 capsid with three surface tyrosine residues changed to phenylalanine (AAV2tYF) vector...

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Source

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Identifiers

PMID: 32414297
DOI: 10.1089/hum.2019.337
PMCID: PMC7404825

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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