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

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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
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  Data: ELP1 gene augmentation restores visual function in a mouse model of familial dysautonomia.
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  Data: <searchLink fieldCode="AU" term="%22Cheng+HC%22">Cheng HC</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Koli+S%22">Koli S</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Lewis+K%22">Lewis K</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Ketchale+S%22">Ketchale S</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Anvarinia+Y%22">Anvarinia Y</searchLink>; Department of Ophthalmology, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Erdoes+M%22">Erdoes M</searchLink>; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Liu+Y%22">Liu Y</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Keiper+CE%22">Keiper CE</searchLink>; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Estelien+R%22">Estelien R</searchLink>; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Chagnon+M%22">Chagnon M</searchLink>; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Vandenberghe+LH%22">Vandenberghe LH</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Gao+D%22">Gao D</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Lefcort+F%22">Lefcort F</searchLink>; Tikun Therapeutics, 20300 Lubar Way, Brooksville, MD, USA.<br /><searchLink fieldCode="AU" term="%22Morini+E%22">Morini E</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Slaugenhaupt+SA%22">Slaugenhaupt SA</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Chekuri+A%22">Chekuri A</searchLink>; 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.
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  Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2026 Aug 05; Vol. 34 (8), pp. 4466-4481. <i>Date of Electronic Publication: </i>2026 Apr 25.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>100890581 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1525-0024 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2215250016%22">15250016 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther <i>Subsets: </i>MEDLINE
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        Value: 10.1016/j.ymthe.2026.04.046
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        Text: English
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