S945L-CFTR molecular dynamics, functional characterization and tezacaftor/ivacaftor efficacy <i>in vivo</i> and <i>in vitro</i> in matched pediatric patient-derived cell models.

Allan, Katelin M; Astore, Miro A; Fawcett, Laura K; Wong, Sharon L; Chen, Po-Chia; Griffith, Renate; Jaffe, Adam; Kuyucak, Serdar; Waters, Shafagh A

S945L-CFTR molecular dynamics, functional characterization and tezacaftor/ivacaftor efficacy in vivo and in vitro in matched pediatric patient-derived cell models.

Allan, Katelin M; Astore, Miro A; Fawcett, Laura K; Wong, Sharon L; Chen, Po-Chia; Griffith, Renate; Jaffe, Adam; Kuyucak, Serdar; Waters, Shafagh A.

Afiliação

Allan KM; School of Clinical Medicine, Discipline of Paediatrics and Child Health, Faculty of Medicine and Health, UNSW Sydney, Sydney, NSW, Australia.
Astore MA; Molecular and Integrative Cystic Fibrosis Research Centre, UNSW Sydney, Sydney, NSW, Australia.
Fawcett LK; School of Physics, The University of Sydney, Sydney, NSW, Australia.
Wong SL; School of Clinical Medicine, Discipline of Paediatrics and Child Health, Faculty of Medicine and Health, UNSW Sydney, Sydney, NSW, Australia.
Chen PC; Molecular and Integrative Cystic Fibrosis Research Centre, UNSW Sydney, Sydney, NSW, Australia.
Griffith R; Department of Respiratory Medicine, Sydney Children's Hospital, Sydney, NSW, Australia.
Jaffe A; School of Clinical Medicine, Discipline of Paediatrics and Child Health, Faculty of Medicine and Health, UNSW Sydney, Sydney, NSW, Australia.
Kuyucak S; Molecular and Integrative Cystic Fibrosis Research Centre, UNSW Sydney, Sydney, NSW, Australia.
Waters SA; School of Physics, The University of Sydney, Sydney, NSW, Australia.

Front Pediatr ; 10: 1062766, 2022.

Article em En | MEDLINE | ID: mdl-36467478

Palavras-chave

CFTR; airway epithelial cell models; cystic fibrosis; modulators; molecular dynamics; personalized medicine

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Revista: Front Pediatr Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Austrália

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