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. |
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| 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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