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The synthetic aminoglycoside ELX-02 induces readthrough of G550X-CFTR producing superfunctional protein that can be further enhanced by CFTR modulators.
Chen, Jianguo; Thrasher, Kari; Fu, Lianwu; Wang, Wei; Aghamohammadzadeh, Soheil; Wen, Hui; Tang, Liping; Keeling, Kim M; Falk Libby, Emily; Bedwell, David M; Rowe, Steven M.
Afiliação
  • Chen J; Department of Medicine, University of Alabama at Birmingham, Birmingham, Alabama, United States.
  • Thrasher K; Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, United States.
  • Fu L; Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham, Birmingham, Alabama, United States.
  • Wang W; Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, United States.
  • Aghamohammadzadeh S; Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham, Birmingham, Alabama, United States.
  • Wen H; Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, United States.
  • Tang L; Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, United States.
  • Keeling KM; Eloxx Pharmaceuticals, Inc., Watertown, Massachusetts, United States.
  • Falk Libby E; Department of Medicine, University of Alabama at Birmingham, Birmingham, Alabama, United States.
  • Bedwell DM; Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, United States.
  • Rowe SM; Department of Medicine, University of Alabama at Birmingham, Birmingham, Alabama, United States.
Am J Physiol Lung Cell Mol Physiol ; 324(6): L756-L770, 2023 06 01.
Article em En | MEDLINE | ID: mdl-37014818
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Regulador de Condutância Transmembrana em Fibrose Cística / Aminoglicosídeos Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Regulador de Condutância Transmembrana em Fibrose Cística / Aminoglicosídeos Idioma: En Ano de publicação: 2023 Tipo de documento: Article