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Atrophin-1 antisense oligonucleotide provides robust protection from pathology in a fully humanized DRPLA model.

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Title: Atrophin-1 antisense oligonucleotide provides robust protection from pathology in a fully humanized DRPLA model.
Authors: Smith VL; Department of Neurology, University of Washington, Seattle, WA 98104, USA., Gidi BZ; Department of Neurology, University of Washington, Seattle, WA 98104, USA., Bragg RM; Department of Neurology, University of Washington, Seattle, WA 98104, USA., Cantle JP; Department of Neurology, University of Washington, Seattle, WA 98104, USA., Ben-Varon A; Department of Psychology, Western Washington University, Bellingham, WA 98225, USA., Noble B; Ionis Pharmaceuticals, Inc., Carlsbad, CA 92010, USA., Prades S; CureDRPLA, 360 Fuman St. Suite 218, Brooklyn, NY 11201, USA.; Ataxia UK, 12 Broadbent Close, London N6 5JW, UK., Compton A; CureDRPLA, 360 Fuman St. Suite 218, Brooklyn, NY 11201, USA.; Ataxia UK, 12 Broadbent Close, London N6 5JW, UK., Greenfield J; CureDRPLA, 360 Fuman St. Suite 218, Brooklyn, NY 11201, USA.; Ataxia UK, 12 Broadbent Close, London N6 5JW, UK., Korecka JA; Division of Movement Disorders and Department of Neurology, Mass General Brigham and Harvard Medical School, Boston, MA 02114, USA., Gemos A; Division of Movement Disorders and Department of Neurology, Mass General Brigham and Harvard Medical School, Boston, MA 02114, USA., Yu T; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA 02115, USA.; Manton Center for Orphan Disease Research, Boston Children's Hospital, Boston, MA 02115, USA.; Department of Pediatrics, Boston Children's Hospital, Boston, MA 02115, USA.; Program in Medical and Population Genetics, Broad Institute of MIT and Harvard, Cambridge, MA 02139, USA., Khurana V; Division of Movement Disorders and Department of Neurology, Mass General Brigham and Harvard Medical School, Boston, MA 02114, USA.; Broad Institute of Harvard and MIT and Harvard Stem Cell Institute, Cambridge, MA 02139, USA., Kordasiewicz HB; Ionis Pharmaceuticals, Inc., Carlsbad, CA 92010, USA., Zhao HT; Ionis Pharmaceuticals, Inc., Carlsbad, CA 92010, USA., Barker-Haliski M; Department of Pharmaceutics, Center for Epilepsy Drug Discovery (CEDD), University of Washington, Seattle, WA 98195, USA., Child DD; Department of Laboratory Medicine and Pathology, University of Washington, Seattle, WA 98104, USA., Carroll JB; Department of Neurology, University of Washington, Seattle, WA 98104, USA.; CureDRPLA, 360 Fuman St. Suite 218, Brooklyn, NY 11201, USA.; Department of Laboratory Medicine and Pathology, University of Washington, Seattle, WA 98104, USA.
Source: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2025 Dec 31; Vol. 37 (1), pp. 102815. Date of Electronic Publication: 2025 Dec 31 (Print Publication: 2026).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101581621 Publication Model: eCollection Cited Medium: Print ISSN: 2162-2531 (Print) Linking ISSN: 21622531 NLM ISO Abbreviation: Mol Ther Nucleic Acids Subsets: PubMed not MEDLINE
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  Data: Atrophin-1 antisense oligonucleotide provides robust protection from pathology in a fully humanized DRPLA model.
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  Data: <searchLink fieldCode="AU" term="%22Smith+VL%22">Smith VL</searchLink>; Department of Neurology, University of Washington, Seattle, WA 98104, USA.<br /><searchLink fieldCode="AU" term="%22Gidi+BZ%22">Gidi BZ</searchLink>; Department of Neurology, University of Washington, Seattle, WA 98104, USA.<br /><searchLink fieldCode="AU" term="%22Bragg+RM%22">Bragg RM</searchLink>; Department of Neurology, University of Washington, Seattle, WA 98104, USA.<br /><searchLink fieldCode="AU" term="%22Cantle+JP%22">Cantle JP</searchLink>; Department of Neurology, University of Washington, Seattle, WA 98104, USA.<br /><searchLink fieldCode="AU" term="%22Ben-Varon+A%22">Ben-Varon A</searchLink>; Department of Psychology, Western Washington University, Bellingham, WA 98225, USA.<br /><searchLink fieldCode="AU" term="%22Noble+B%22">Noble B</searchLink>; Ionis Pharmaceuticals, Inc., Carlsbad, CA 92010, USA.<br /><searchLink fieldCode="AU" term="%22Prades+S%22">Prades S</searchLink>; CureDRPLA, 360 Fuman St. Suite 218, Brooklyn, NY 11201, USA.; Ataxia UK, 12 Broadbent Close, London N6 5JW, UK.<br /><searchLink fieldCode="AU" term="%22Compton+A%22">Compton A</searchLink>; CureDRPLA, 360 Fuman St. Suite 218, Brooklyn, NY 11201, USA.; Ataxia UK, 12 Broadbent Close, London N6 5JW, UK.<br /><searchLink fieldCode="AU" term="%22Greenfield+J%22">Greenfield J</searchLink>; CureDRPLA, 360 Fuman St. Suite 218, Brooklyn, NY 11201, USA.; Ataxia UK, 12 Broadbent Close, London N6 5JW, UK.<br /><searchLink fieldCode="AU" term="%22Korecka+JA%22">Korecka JA</searchLink>; Division of Movement Disorders and Department of Neurology, Mass General Brigham and Harvard Medical School, Boston, MA 02114, USA.<br /><searchLink fieldCode="AU" term="%22Gemos+A%22">Gemos A</searchLink>; Division of Movement Disorders and Department of Neurology, Mass General Brigham and Harvard Medical School, Boston, MA 02114, USA.<br /><searchLink fieldCode="AU" term="%22Yu+T%22">Yu T</searchLink>; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA 02115, USA.; Manton Center for Orphan Disease Research, Boston Children's Hospital, Boston, MA 02115, USA.; Department of Pediatrics, Boston Children's Hospital, Boston, MA 02115, USA.; Program in Medical and Population Genetics, Broad Institute of MIT and Harvard, Cambridge, MA 02139, USA.<br /><searchLink fieldCode="AU" term="%22Khurana+V%22">Khurana V</searchLink>; Division of Movement Disorders and Department of Neurology, Mass General Brigham and Harvard Medical School, Boston, MA 02114, USA.; Broad Institute of Harvard and MIT and Harvard Stem Cell Institute, Cambridge, MA 02139, USA.<br /><searchLink fieldCode="AU" term="%22Kordasiewicz+HB%22">Kordasiewicz HB</searchLink>; Ionis Pharmaceuticals, Inc., Carlsbad, CA 92010, USA.<br /><searchLink fieldCode="AU" term="%22Zhao+HT%22">Zhao HT</searchLink>; Ionis Pharmaceuticals, Inc., Carlsbad, CA 92010, USA.<br /><searchLink fieldCode="AU" term="%22Barker-Haliski+M%22">Barker-Haliski M</searchLink>; Department of Pharmaceutics, Center for Epilepsy Drug Discovery (CEDD), University of Washington, Seattle, WA 98195, USA.<br /><searchLink fieldCode="AU" term="%22Child+DD%22">Child DD</searchLink>; Department of Laboratory Medicine and Pathology, University of Washington, Seattle, WA 98104, USA.<br /><searchLink fieldCode="AU" term="%22Carroll+JB%22">Carroll JB</searchLink>; Department of Neurology, University of Washington, Seattle, WA 98104, USA.; CureDRPLA, 360 Fuman St. Suite 218, Brooklyn, NY 11201, USA.; Department of Laboratory Medicine and Pathology, University of Washington, Seattle, WA 98104, USA.
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  Data: <searchLink fieldCode="JN" term="%22101581621%22">Molecular therapy. Nucleic acids</searchLink> [Mol Ther Nucleic Acids] 2025 Dec 31; Vol. 37 (1), pp. 102815. <i>Date of Electronic Publication: </i>2025 Dec 31 (<i>Print Publication: </i>2026).
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