Development of an AAV-based gene therapy for the ocular phenotype of Friedreich's ataxia.

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Bibliographic Details
Title: Development of an AAV-based gene therapy for the ocular phenotype of Friedreich's ataxia.
Authors: Tang H; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Gupte S; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Xu E; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Calabro KR; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Friend H; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Crosson SM; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Fajardo D; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Kostamo Z; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Zhang H; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Peterson JJ; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Lin F; Department of Ophthalmology, Emory University, Atlanta, GA, USA., Kozmik Z; Institute of Molecular Genetics, Academy of Sciences of the Czech Republic, Prague, Czech Republic., Lutz CM; Rare and Orphan Disease Center, The Jackson Laboratory, Bar Harbor, ME, USA., Boye SL; Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA., Boye SE; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA. Electronic address: shaire@ufl.edu.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 Feb 04; Vol. 34 (2), pp. 771-788. Date of Electronic Publication: 2025 Oct 24.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1525-0024
DOI:10.1016/j.ymthe.2025.10.048