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An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion.
Keller, Caroline Gubser; Shin, Youngah; Monteys, Alex Mas; Renaud, Nicole; Beibel, Martin; Teider, Natalia; Peters, Thomas; Faller, Thomas; St-Cyr, Sophie; Knehr, Judith; Roma, Guglielmo; Reyes, Alejandro; Hild, Marc; Lukashev, Dmitriy; Theil, Diethilde; Dales, Natalie; Cha, Jang-Ho; Borowsky, Beth; Dolmetsch, Ricardo; Davidson, Beverly L; Sivasankaran, Rajeev.
Afiliação
  • 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.
  • Renaud N; Department of Pathology and Laboratory Medicine, The Perelman School of Medicine, The University of Pennsylvania, Philadelphia, PA, USA.
  • Beibel M; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.
  • Teider N; Novartis Institutes for Biomedical Research, Basel, Switzerland.
  • Peters T; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.
  • Faller T; Novartis Institutes for Biomedical Research, Basel, Switzerland.
  • St-Cyr S; Novartis Institutes for Biomedical Research, Basel, Switzerland.
  • Knehr J; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • 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, Basel, Switzerland.
  • Lukashev D; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.
  • Theil D; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.
  • Dales N; Novartis Institutes for Biomedical Research, Basel, Switzerland.
  • Cha JH; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.
  • Borowsky B; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.
  • Dolmetsch R; Novartis Pharmaceuticals, East Hanover, NJ, USA.
  • Davidson BL; Novartis Institutes for Biomedical Research, Cambridge, MA, USA.
  • Sivasankaran R; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. davidsonbl@chop.edu.
Nat Commun ; 13(1): 1150, 2022 03 03.
Article em En | MEDLINE | ID: mdl-35241644
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Huntington Limite: Animals / Humans Idioma: En Revista: Nat Commun Assunto da revista: BIOLOGIA / CIENCIA Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Suíça
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Huntington Limite: Animals / Humans Idioma: En Revista: Nat Commun Assunto da revista: BIOLOGIA / CIENCIA Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Suíça