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Peripheral neuron phenotypes of familial dysautonomia are rescued by AAV-mediated gene therapy.

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Title: Peripheral neuron phenotypes of familial dysautonomia are rescued by AAV-mediated gene therapy.
Authors: Wu HF; Center for Molecular Medicine, University of Georgia, Athens, GA, USA.; Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA, USA., Saini T; Center for Molecular Medicine, University of Georgia, Athens, GA, USA.; Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA, USA., Art J; Center for Molecular Medicine, University of Georgia, Athens, GA, USA.; Biomedical and Translational Sciences Institute, Neuroscience Program, University of Georgia, Athens, GA, 30602, USA., Delaney W; Center for Molecular Medicine, University of Georgia, Athens, GA, USA., Lefcort F; Tikun Therapeutics and Familial Dysautonomia Foundation, New York, NY, USA., Zeltner N; Center for Molecular Medicine, University of Georgia, Athens, GA, USA. nadja.zeltner@uga.edu.; Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA, USA. nadja.zeltner@uga.edu.; Department of Cellular Biology, University of Georgia, Athens, GA, USA. nadja.zeltner@uga.edu.
Source: Clinical autonomic research : official journal of the Clinical Autonomic Research Society [Clin Auton Res] 2026 Jun; Vol. 36 (3), pp. 417-421. Date of Electronic Publication: 2026 Apr 09.
Publication Type: Letter
Journal Info: Publisher: Steinkopff Country of Publication: Germany NLM ID: 9106549 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1619-1560 (Electronic) Linking ISSN: 09599851 NLM ISO Abbreviation: Clin Auton Res Subsets: MEDLINE; In Process
Database: MEDLINE Ultimate
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  Data: Peripheral neuron phenotypes of familial dysautonomia are rescued by AAV-mediated gene therapy.
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  Data: <searchLink fieldCode="AU" term="%22Wu+HF%22">Wu HF</searchLink>; Center for Molecular Medicine, University of Georgia, Athens, GA, USA.; Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA, USA.<br /><searchLink fieldCode="AU" term="%22Saini+T%22">Saini T</searchLink>; Center for Molecular Medicine, University of Georgia, Athens, GA, USA.; Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA, USA.<br /><searchLink fieldCode="AU" term="%22Art+J%22">Art J</searchLink>; Center for Molecular Medicine, University of Georgia, Athens, GA, USA.; Biomedical and Translational Sciences Institute, Neuroscience Program, University of Georgia, Athens, GA, 30602, USA.<br /><searchLink fieldCode="AU" term="%22Delaney+W%22">Delaney W</searchLink>; Center for Molecular Medicine, University of Georgia, Athens, GA, USA.<br /><searchLink fieldCode="AU" term="%22Lefcort+F%22">Lefcort F</searchLink>; Tikun Therapeutics and Familial Dysautonomia Foundation, New York, NY, USA.<br /><searchLink fieldCode="AU" term="%22Zeltner+N%22">Zeltner N</searchLink>; Center for Molecular Medicine, University of Georgia, Athens, GA, USA. nadja.zeltner@uga.edu.; Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA, USA. nadja.zeltner@uga.edu.; Department of Cellular Biology, University of Georgia, Athens, GA, USA. nadja.zeltner@uga.edu.
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  Data: <searchLink fieldCode="JN" term="%229106549%22">Clinical autonomic research : official journal of the Clinical Autonomic Research Society</searchLink> [Clin Auton Res] 2026 Jun; Vol. 36 (3), pp. 417-421. <i>Date of Electronic Publication: </i>2026 Apr 09.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Steinkopff%22">Steinkopff </searchLink><i>Country of Publication: </i>Germany <i>NLM ID: </i>9106549 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1619-1560 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2209599851%22">09599851 </searchLink><i>NLM ISO Abbreviation: </i>Clin Auton Res <i>Subsets: </i>MEDLINE; In Process
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      – Type: doi
        Value: 10.1007/s10286-026-01206-3
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      – Code: eng
        Text: English
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        StartPage: 417
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      – TitleFull: Peripheral neuron phenotypes of familial dysautonomia are rescued by AAV-mediated gene therapy.
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            NameFull: Saini T
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              Text: 2026 Jun
              Type: published
              Y: 2026
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              Value: 36
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            – TitleFull: Clinical autonomic research : official journal of the Clinical Autonomic Research Society
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