ELP1 gene augmentation restores visual function in a mouse model of familial dysautonomia.

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Bibliographic Details
Title: ELP1 gene augmentation restores visual function in a mouse model of familial dysautonomia.
Authors: Cheng HC; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA; Department of Ophthalmology, Taipei Veterans General Hospital, Taipei, Taiwan; Department of Ophthalmology, School of Medicine, National Yang Ming Chiao Tung University, Taipei, Taiwan., Koli S; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA; Department of Ophthalmology, Harvard Medical School, Boston, MA, USA., Lewis K; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA; Department of Ophthalmology, Harvard Medical School, Boston, MA, USA., Ketchale S; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA; Department of Ophthalmology, Harvard Medical School, Boston, MA, USA., Anvarinia Y; Department of Ophthalmology, Harvard Medical School, Boston, MA, USA., Erdoes M; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA., Liu Y; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA., Keiper CE; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA., Estelien R; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA., Chagnon M; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA., Vandenberghe LH; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA; Department of Ophthalmology, Harvard Medical School, Boston, MA, USA., Gao D; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA., Lefcort F; Tikun Therapeutics, 20300 Lubar Way, Brooksville, MD, USA., Morini E; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA., Slaugenhaupt SA; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA., Chekuri A; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA; Department of Ophthalmology, Harvard Medical School, Boston, MA, USA. Electronic address: anil_chekuri@meei.harvard.edu.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 Aug 05; Vol. 34 (8), pp. 4466-4481. Date of Electronic Publication: 2026 Apr 25.
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.2026.04.046