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The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy.

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Title: The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy.
Authors: d'Ydewalle C; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA., Ramos DM; Department of Neuroscience, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA., Pyles NJ; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA., Ng SY; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA 02138, USA., Gorz M; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA., Pilato CM; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA., Ling K; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Kong L; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA., Ward AJ; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, 500 Main Street, Cambridge, MA 02142, USA., Rubin LL; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA 02138, USA., Rigo F; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Bennett CF; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Sumner CJ; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA; Department of Neuroscience, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA. Electronic address: csumner1@jhmi.edu.
Source: Neuron [Neuron] 2017 Jan 04; Vol. 93 (1), pp. 66-79. Date of Electronic Publication: 2016 Dec 22.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 8809320 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4199 (Electronic) Linking ISSN: 08966273 NLM ISO Abbreviation: Neuron Subsets: MEDLINE
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  Data: The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy.
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  Data: <searchLink fieldCode="AU" term="%22d'Ydewalle+C%22">d'Ydewalle C</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.<br /><searchLink fieldCode="AU" term="%22Ramos+DM%22">Ramos DM</searchLink>; Department of Neuroscience, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.<br /><searchLink fieldCode="AU" term="%22Pyles+NJ%22">Pyles NJ</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.<br /><searchLink fieldCode="AU" term="%22Ng+SY%22">Ng SY</searchLink>; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA 02138, USA.<br /><searchLink fieldCode="AU" term="%22Gorz+M%22">Gorz M</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.<br /><searchLink fieldCode="AU" term="%22Pilato+CM%22">Pilato CM</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.<br /><searchLink fieldCode="AU" term="%22Ling+K%22">Ling K</searchLink>; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.<br /><searchLink fieldCode="AU" term="%22Kong+L%22">Kong L</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.<br /><searchLink fieldCode="AU" term="%22Ward+AJ%22">Ward AJ</searchLink>; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, 500 Main Street, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Rubin+LL%22">Rubin LL</searchLink>; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA 02138, USA.<br /><searchLink fieldCode="AU" term="%22Rigo+F%22">Rigo F</searchLink>; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.<br /><searchLink fieldCode="AU" term="%22Bennett+CF%22">Bennett CF</searchLink>; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.<br /><searchLink fieldCode="AU" term="%22Sumner+CJ%22">Sumner CJ</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA; Department of Neuroscience, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA. Electronic address: csumner1@jhmi.edu.
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  Data: <searchLink fieldCode="JN" term="%228809320%22">Neuron</searchLink> [Neuron] 2017 Jan 04; Vol. 93 (1), pp. 66-79. <i>Date of Electronic Publication: </i>2016 Dec 22.
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