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An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion.

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Title: An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion.
Authors: Keller CG; Novartis Institutes for Biomedical Research, Basel, Switzerland., Shin Y; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Monteys AM; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Department of Pathology and Laboratory Medicine, The Perelman School of Medicine, The University of Pennsylvania, Philadelphia, PA, USA., Renaud N; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Beibel M; Novartis Institutes for Biomedical Research, Basel, Switzerland., Teider N; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Peters T; Novartis Institutes for Biomedical Research, Basel, Switzerland., Faller T; Novartis Institutes for Biomedical Research, Basel, Switzerland., St-Cyr S; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Knehr J; Novartis Institutes for Biomedical Research, Basel, Switzerland., Roma G; Novartis Institutes for Biomedical Research, Basel, Switzerland., Reyes A; Novartis Institutes for Biomedical Research, Basel, Switzerland., Hild M; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Lukashev D; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Theil D; Novartis Institutes for Biomedical Research, Basel, Switzerland., Dales N; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Cha JH; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Borowsky B; Novartis Pharmaceuticals, East Hanover, NJ, USA., Dolmetsch R; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Davidson BL; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. davidsonbl@chop.edu.; Department of Pathology and Laboratory Medicine, The Perelman School of Medicine, The University of Pennsylvania, Philadelphia, PA, USA. davidsonbl@chop.edu., Sivasankaran R; Novartis Institutes for Biomedical Research, Cambridge, MA, USA. rajeev.sivasankaran@novartis.com.
Source: Nature communications [Nat Commun] 2022 Mar 03; Vol. 13 (1), pp. 1150. Date of Electronic Publication: 2022 Mar 03.
Publication Type: Journal Article; Clinical Trial, Phase II; Clinical Trial, Phase I
Journal Info: Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion.
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  Data: <searchLink fieldCode="AU" term="%22Keller+CG%22">Keller CG</searchLink>; Novartis Institutes for Biomedical Research, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Shin+Y%22">Shin Y</searchLink>; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Monteys+AM%22">Monteys AM</searchLink>; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Department of Pathology and Laboratory Medicine, The Perelman School of Medicine, The University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Renaud+N%22">Renaud N</searchLink>; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Beibel+M%22">Beibel M</searchLink>; Novartis Institutes for Biomedical Research, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Teider+N%22">Teider N</searchLink>; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Peters+T%22">Peters T</searchLink>; Novartis Institutes for Biomedical Research, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Faller+T%22">Faller T</searchLink>; Novartis Institutes for Biomedical Research, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22St-Cyr+S%22">St-Cyr S</searchLink>; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Knehr+J%22">Knehr J</searchLink>; Novartis Institutes for Biomedical Research, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Roma+G%22">Roma G</searchLink>; Novartis Institutes for Biomedical Research, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Reyes+A%22">Reyes A</searchLink>; Novartis Institutes for Biomedical Research, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Hild+M%22">Hild M</searchLink>; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Lukashev+D%22">Lukashev D</searchLink>; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Theil+D%22">Theil D</searchLink>; Novartis Institutes for Biomedical Research, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Dales+N%22">Dales N</searchLink>; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Cha+JH%22">Cha JH</searchLink>; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Borowsky+B%22">Borowsky B</searchLink>; Novartis Pharmaceuticals, East Hanover, NJ, USA.<br /><searchLink fieldCode="AU" term="%22Dolmetsch+R%22">Dolmetsch R</searchLink>; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Davidson+BL%22">Davidson BL</searchLink>; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. davidsonbl@chop.edu.; Department of Pathology and Laboratory Medicine, The Perelman School of Medicine, The University of Pennsylvania, Philadelphia, PA, USA. davidsonbl@chop.edu.<br /><searchLink fieldCode="AU" term="%22Sivasankaran+R%22">Sivasankaran R</searchLink>; Novartis Institutes for Biomedical Research, Cambridge, MA, USA. rajeev.sivasankaran@novartis.com.
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  Data: <searchLink fieldCode="JN" term="%22101528555%22">Nature communications</searchLink> [Nat Commun] 2022 Mar 03; Vol. 13 (1), pp. 1150. <i>Date of Electronic Publication: </i>2022 Mar 03.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Nature+Pub%2E+Group%22">Nature Pub. Group </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>101528555 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2041-1723 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2220411723%22">20411723 </searchLink><i>NLM ISO Abbreviation: </i>Nat Commun <i>Subsets: </i>MEDLINE
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