Selective targeting of mutant huntingtin intron 1 improves rescue provided by antisense oligonucleotides in Huntington's disease mice.

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Title: Selective targeting of mutant huntingtin intron 1 improves rescue provided by antisense oligonucleotides in Huntington's disease mice.
Authors: Bragg RM; Department of Neurology, University of Washington, Seattle, WA 98104, USA., Landles C; Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Smith EJ; Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Osborne GF; Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Mathews EW; Department of Neurology, University of Washington, Seattle, WA 98104, USA., Cantle JP; Department of Neurology, University of Washington, Seattle, WA 98104, USA., Bates GP; Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK., Carroll JB; Department of Neurology, University of Washington, Seattle, WA 98104, USA.
Source: Science translational medicine [Sci Transl Med] 2026 Mar 18; Vol. 18 (841), pp. eadv0702. Date of Electronic Publication: 2026 Mar 18.
Publication Type: Journal Article
Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic 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
DOI:10.1126/scitranslmed.adv0702