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Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models.

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Title: Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models.
Authors: Morelli KH; The Jackson Laboratory, Bar Harbor, Maine, USA.; Graduate School of Biomedical Science and Engineering, University of Maine, Orono, Maine, USA., Griffin LB; Program in Cellular and Molecular Biology, and.; Medical Scientist Training Program, University of Michigan, Ann Arbor, Michigan, USA., Pyne NK; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Wallace LM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Fowler AM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Oprescu SN; Department of Human Genetics, University of Michigan, Ann Arbor, Michigan, USA., Takase R; Department of Biochemistry and Molecular Biochemistry, Thomas Jefferson University, Philadelphia, Pennsylvania, USA., Wei N; Department of Molecular Medicine, The Scripps Research Institute, La Jolla, California, USA., Meyer-Schuman R; Department of Human Genetics, University of Michigan, Ann Arbor, Michigan, USA., Mellacheruvu D; Department of Pathology, and.; Department of Computational Medicine and Bioinformatics, University of Michigan, Ann Arbor, Michigan, USA., Kitzman JO; Department of Human Genetics, University of Michigan, Ann Arbor, Michigan, USA., Kocen SG; The Jackson Laboratory, Bar Harbor, Maine, USA., Hines TJ; The Jackson Laboratory, Bar Harbor, Maine, USA., Spaulding EL; The Jackson Laboratory, Bar Harbor, Maine, USA.; Graduate School of Biomedical Science and Engineering, University of Maine, Orono, Maine, USA., Lupski JR; Department of Molecular and Human Genetics, and.; Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.; Texas Children's Hospital, Houston, Texas, USA., Nesvizhskii A; Department of Pathology, and.; Department of Computational Medicine and Bioinformatics, University of Michigan, Ann Arbor, Michigan, USA., Mancias P; Department of Pediatrics, McGovern Medical School, The University of Texas Health Science Center at Houston, and Children's Memorial Hermann Hospital, Houston, Texas, USA., Butler IJ; Department of Pediatrics, McGovern Medical School, The University of Texas Health Science Center at Houston, and Children's Memorial Hermann Hospital, Houston, Texas, USA., Yang XL; Department of Molecular Medicine, The Scripps Research Institute, La Jolla, California, USA., Hou YM; Department of Biochemistry and Molecular Biochemistry, Thomas Jefferson University, Philadelphia, Pennsylvania, USA., Antonellis A; Program in Cellular and Molecular Biology, and.; Department of Human Genetics, University of Michigan, Ann Arbor, Michigan, USA., Harper SQ; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA., Burgess RW; The Jackson Laboratory, Bar Harbor, Maine, USA.; Graduate School of Biomedical Science and Engineering, University of Maine, Orono, Maine, USA.
Source: The Journal of clinical investigation [J Clin Invest] 2019 Dec 02; Vol. 129 (12), pp. 5568-5583.
Publication Type: Journal Article; Research Support, N.I.H., Extramural
Journal Info: Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 7802877 Publication Model: Print Cited Medium: Internet ISSN: 1558-8238 (Electronic) Linking ISSN: 00219738 NLM ISO Abbreviation: J Clin Invest Subsets: MEDLINE
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  Data: Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models.
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  Data: <searchLink fieldCode="AU" term="%22Morelli+KH%22">Morelli KH</searchLink>; The Jackson Laboratory, Bar Harbor, Maine, USA.; Graduate School of Biomedical Science and Engineering, University of Maine, Orono, Maine, USA.<br /><searchLink fieldCode="AU" term="%22Griffin+LB%22">Griffin LB</searchLink>; Program in Cellular and Molecular Biology, and.; Medical Scientist Training Program, University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Pyne+NK%22">Pyne NK</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Wallace+LM%22">Wallace LM</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Fowler+AM%22">Fowler AM</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Oprescu+SN%22">Oprescu SN</searchLink>; Department of Human Genetics, University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Takase+R%22">Takase R</searchLink>; Department of Biochemistry and Molecular Biochemistry, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.<br /><searchLink fieldCode="AU" term="%22Wei+N%22">Wei N</searchLink>; Department of Molecular Medicine, The Scripps Research Institute, La Jolla, California, USA.<br /><searchLink fieldCode="AU" term="%22Meyer-Schuman+R%22">Meyer-Schuman R</searchLink>; Department of Human Genetics, University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Mellacheruvu+D%22">Mellacheruvu D</searchLink>; Department of Pathology, and.; Department of Computational Medicine and Bioinformatics, University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Kitzman+JO%22">Kitzman JO</searchLink>; Department of Human Genetics, University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Kocen+SG%22">Kocen SG</searchLink>; The Jackson Laboratory, Bar Harbor, Maine, USA.<br /><searchLink fieldCode="AU" term="%22Hines+TJ%22">Hines TJ</searchLink>; The Jackson Laboratory, Bar Harbor, Maine, USA.<br /><searchLink fieldCode="AU" term="%22Spaulding+EL%22">Spaulding EL</searchLink>; The Jackson Laboratory, Bar Harbor, Maine, USA.; Graduate School of Biomedical Science and Engineering, University of Maine, Orono, Maine, USA.<br /><searchLink fieldCode="AU" term="%22Lupski+JR%22">Lupski JR</searchLink>; Department of Molecular and Human Genetics, and.; Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.; Texas Children's Hospital, Houston, Texas, USA.<br /><searchLink fieldCode="AU" term="%22Nesvizhskii+A%22">Nesvizhskii A</searchLink>; Department of Pathology, and.; Department of Computational Medicine and Bioinformatics, University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Mancias+P%22">Mancias P</searchLink>; Department of Pediatrics, McGovern Medical School, The University of Texas Health Science Center at Houston, and Children's Memorial Hermann Hospital, Houston, Texas, USA.<br /><searchLink fieldCode="AU" term="%22Butler+IJ%22">Butler IJ</searchLink>; Department of Pediatrics, McGovern Medical School, The University of Texas Health Science Center at Houston, and Children's Memorial Hermann Hospital, Houston, Texas, USA.<br /><searchLink fieldCode="AU" term="%22Yang+XL%22">Yang XL</searchLink>; Department of Molecular Medicine, The Scripps Research Institute, La Jolla, California, USA.<br /><searchLink fieldCode="AU" term="%22Hou+YM%22">Hou YM</searchLink>; Department of Biochemistry and Molecular Biochemistry, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.<br /><searchLink fieldCode="AU" term="%22Antonellis+A%22">Antonellis A</searchLink>; Program in Cellular and Molecular Biology, and.; Department of Human Genetics, University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Harper+SQ%22">Harper SQ</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Burgess+RW%22">Burgess RW</searchLink>; The Jackson Laboratory, Bar Harbor, Maine, USA.; Graduate School of Biomedical Science and Engineering, University of Maine, Orono, Maine, USA.
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