Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype

Molecular Genetics & Genomic Medicine

Giovanni Taccetti¹  Chiara Castellani¹  Vito Terlizzi¹  Beatrice Ferrari¹  Giuseppe Castaldo²  Felice Amato²  Luis J. V. Galietta³ 

[1] Cystic Fibrosis Regional Reference Center,, Department of Paediatric Medicine, Anna Meyer Children's University, Florence, Italy;Department of Molecular Medicine and Medical Biotechnology, University of Naples Federico II;CEINGE – Advanced Biotechnologies, Naples, Italy;Telethon Institute of Genetics and Medicine (TIGEM, Pozzuoli, Italy);Department of Translational Medical Sciences, University of Naples Federico II, Napoli, Italy;

关键词: children;    cystic fibrosis;    lumacaftor/ivacaftor;    nasal brushing;   

DOI  :  10.1002/mgg3.1656

来源: Wiley

【 授权许可】

CC BY