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
Database: MEDLINE Ultimate
Description
ISSN:2162-2531
DOI:10.1016/j.omtn.2025.102815