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Peripherally administered androgen receptor-targeted antisense oligonucleotide rescues spinal pathology in a murine SBMA model.

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Title: Peripherally administered androgen receptor-targeted antisense oligonucleotide rescues spinal pathology in a murine SBMA model.
Authors: Lee C; Interdepartmental Neuroscience Program, Department of Neuroscience, Yale School of Medicine, New Haven, Connecticut, USA., Yu Z; Department of Pathology and., Kuo CJ; Department of Pathology and.; Cellular and Molecular Biology Graduate Program, Medical Scientist Training Program, University of Michigan Medical School, Ann Arbor, Michigan, USA., Tejwani L; Interdepartmental Neuroscience Program, Department of Neuroscience, Yale School of Medicine, New Haven, Connecticut, USA., Grijalva RM; Interdepartmental Neuroscience Program, Department of Neuroscience, Yale School of Medicine, New Haven, Connecticut, USA., Bae E; Department of Genetics, Yale School of Medicine, New Haven, Connecticut, USA., Zhao HT; Ionis Pharmaceuticals Inc., Carlsbad, California, USA., Lim J; Interdepartmental Neuroscience Program, Department of Neuroscience, Yale School of Medicine, New Haven, Connecticut, USA.; Department of Genetics, Yale School of Medicine, New Haven, Connecticut, USA.; Department of Neuroscience, Yale School of Medicine, New Haven, Connecticut, USA., Lieberman AP; Department of Pathology and.
Source: The Journal of clinical investigation [J Clin Invest] 2025 Aug 28; Vol. 135 (21). Date of Electronic Publication: 2025 Aug 28 (Print Publication: 2025).
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 7802877 Publication Model: eCollection Cited Medium: Internet ISSN: 1558-8238 (Electronic) Linking ISSN: 00219738 NLM ISO Abbreviation: J Clin Invest Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Peripherally administered androgen receptor-targeted antisense oligonucleotide rescues spinal pathology in a murine SBMA model.
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  Data: <searchLink fieldCode="AU" term="%22Lee+C%22">Lee C</searchLink>; Interdepartmental Neuroscience Program, Department of Neuroscience, Yale School of Medicine, New Haven, Connecticut, USA.<br /><searchLink fieldCode="AU" term="%22Yu+Z%22">Yu Z</searchLink>; Department of Pathology and.<br /><searchLink fieldCode="AU" term="%22Kuo+CJ%22">Kuo CJ</searchLink>; Department of Pathology and.; Cellular and Molecular Biology Graduate Program, Medical Scientist Training Program, University of Michigan Medical School, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Tejwani+L%22">Tejwani L</searchLink>; Interdepartmental Neuroscience Program, Department of Neuroscience, Yale School of Medicine, New Haven, Connecticut, USA.<br /><searchLink fieldCode="AU" term="%22Grijalva+RM%22">Grijalva RM</searchLink>; Interdepartmental Neuroscience Program, Department of Neuroscience, Yale School of Medicine, New Haven, Connecticut, USA.<br /><searchLink fieldCode="AU" term="%22Bae+E%22">Bae E</searchLink>; Department of Genetics, Yale School of Medicine, New Haven, Connecticut, USA.<br /><searchLink fieldCode="AU" term="%22Zhao+HT%22">Zhao HT</searchLink>; Ionis Pharmaceuticals Inc., Carlsbad, California, USA.<br /><searchLink fieldCode="AU" term="%22Lim+J%22">Lim J</searchLink>; Interdepartmental Neuroscience Program, Department of Neuroscience, Yale School of Medicine, New Haven, Connecticut, USA.; Department of Genetics, Yale School of Medicine, New Haven, Connecticut, USA.; Department of Neuroscience, Yale School of Medicine, New Haven, Connecticut, USA.<br /><searchLink fieldCode="AU" term="%22Lieberman+AP%22">Lieberman AP</searchLink>; Department of Pathology and.
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  Data: <searchLink fieldCode="JN" term="%227802877%22">The Journal of clinical investigation</searchLink> [J Clin Invest] 2025 Aug 28; Vol. 135 (21). <i>Date of Electronic Publication: </i>2025 Aug 28 (<i>Print Publication: </i>2025).
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22American+Society+for+Clinical+Investigation%22">American Society for Clinical Investigation </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>7802877 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Internet <i>ISSN: </i>1558-8238 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2200219738%22">00219738 </searchLink><i>NLM ISO Abbreviation: </i>J Clin Invest <i>Subsets: </i>MEDLINE
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        Value: 10.1172/JCI182955
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        Text: English
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      – TitleFull: Peripherally administered androgen receptor-targeted antisense oligonucleotide rescues spinal pathology in a murine SBMA model.
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            – D: 28
              M: 08
              Text: 2025 Aug 28
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