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Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7.

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Title: Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7.
Authors: Niu C; Department of Neurology, Duke University School of Medicine, Durham, NC 27710, USA., Prakash TP; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA., Kim A; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA., Quach JL; Department of Ophthalmology, University of California, San Diego, La Jolla, CA 92093, USA., Huryn LA; National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA., Yang Y; Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA., Lopez E; Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA., Jazayeri A; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA., Hung G; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA., Sopher BL; Department of Neurology, University of Washington, Seattle, WA 98195, USA., Brooks BP; National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA., Swayze EE; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA., Bennett CF; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA., La Spada AR; Department of Neurology, Duke University School of Medicine, Durham, NC 27710, USA. al.laspada@duke.edu.; Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.; Department of Neurobiology, Duke University School of Medicine, Durham, NC 27710, USA.; Department of Cell Biology, Duke University School of Medicine, Durham, NC 27710, USA.; Duke Center for Neurodegeneration and Neurotherapeutics, Duke University School of Medicine, Durham, NC 27710, USA.
Source: Science translational medicine [Sci Transl Med] 2018 Oct 31; Vol. 10 (465).
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7.
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  Data: <searchLink fieldCode="AU" term="%22Niu+C%22">Niu C</searchLink>; Department of Neurology, Duke University School of Medicine, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Prakash+TP%22">Prakash TP</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA.<br /><searchLink fieldCode="AU" term="%22Kim+A%22">Kim A</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA.<br /><searchLink fieldCode="AU" term="%22Quach+JL%22">Quach JL</searchLink>; Department of Ophthalmology, University of California, San Diego, La Jolla, CA 92093, USA.<br /><searchLink fieldCode="AU" term="%22Huryn+LA%22">Huryn LA</searchLink>; National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.<br /><searchLink fieldCode="AU" term="%22Yang+Y%22">Yang Y</searchLink>; Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.<br /><searchLink fieldCode="AU" term="%22Lopez+E%22">Lopez E</searchLink>; Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.<br /><searchLink fieldCode="AU" term="%22Jazayeri+A%22">Jazayeri A</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA.<br /><searchLink fieldCode="AU" term="%22Hung+G%22">Hung G</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA.<br /><searchLink fieldCode="AU" term="%22Sopher+BL%22">Sopher BL</searchLink>; Department of Neurology, University of Washington, Seattle, WA 98195, USA.<br /><searchLink fieldCode="AU" term="%22Brooks+BP%22">Brooks BP</searchLink>; National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.<br /><searchLink fieldCode="AU" term="%22Swayze+EE%22">Swayze EE</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA.<br /><searchLink fieldCode="AU" term="%22Bennett+CF%22">Bennett CF</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA 92008, USA.<br /><searchLink fieldCode="AU" term="%22La+Spada+AR%22">La Spada AR</searchLink>; Department of Neurology, Duke University School of Medicine, Durham, NC 27710, USA. al.laspada@duke.edu.; Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.; Department of Neurobiology, Duke University School of Medicine, Durham, NC 27710, USA.; Department of Cell Biology, Duke University School of Medicine, Durham, NC 27710, USA.; Duke Center for Neurodegeneration and Neurotherapeutics, Duke University School of Medicine, Durham, NC 27710, USA.
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  Data: <searchLink fieldCode="JN" term="%22101505086%22">Science translational medicine</searchLink> [Sci Transl Med] 2018 Oct 31; Vol. 10 (465).
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