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. |
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| 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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| ISSN: | 1946-6242 |
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| DOI: | 10.1126/scitranslmed.aap8677 |
