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Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia.

Autor: Morini E; Center for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital Research Institute and Harvard Medical School, Boston, MA, USA. Electronic address: emorini@mgh.harvard.edu., Chekuri A; Center for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital Research Institute and Harvard Medical School, Boston, MA, USA; Grousbeck Gene Therapy Center, Schepens Eye Research Institute and Massachusetts Eye and Ear Infirmary, Boston, MA, USA., Logan EM; Center for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA., Bolduc JM; Center for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA., Kirchner EG; Center for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA., Salani M; Center for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA., Krauson AJ; Center for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA., Narasimhan J; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Gabbeta V; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Grover S; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Dakka A; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Mollin A; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Jung SP; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Zhao X; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Zhang N; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Zhang S; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Arnold M; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Woll MG; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Naryshkin NA; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Weetall M; PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA., Slaugenhaupt SA; Center for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital Research Institute and Harvard Medical School, Boston, MA, USA. Electronic address: slaugenhaupt@mgh.harvard.edu.

Publikováno v: American journal of human genetics [Am J Hum Genet] 2023 Mar 02; Vol. 110 (3), pp. 531-547. Date of Electronic Publication: 2023 Feb 20.