In vitro modulator responsiveness of 655 CFTR variants found in people with cystic fibrosis-Reference-Cited by-同舟云学术

In vitro modulator responsiveness of 655 CFTR variants found in people with cystic fibrosis

Published:2024-07 Issue:4 Volume:23 Page:664-675
ISSN:1569-1993
Container-title:Journal of Cystic Fibrosis
language:en
Short-container-title:Journal of Cystic Fibrosis

Author:

Bihler Hermann,Sivachenko Andrey,Millen Linda,Bhatt Priyanka^ORCID,Patel Amita Thakerar,Chin Justin,Bailey Violaine,Musisi Isaac,LaPan André^ORCID,Allaire Normand E.,Conte Joshua^ORCID,Simon Noah R.,Magaret Amalia S.^ORCID,Raraigh Karen S.^ORCID,Cutting Garry R.^ORCID,Skach William R.,Bridges Robert J.,Thomas Philip J.^ORCID,Mense Martin^ORCID

Funder

Cystic Fibrosis Foundation

Publisher

Elsevier BV

Reference44 articles.

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3. Cystic Fibrosis;Shteinberg;Lancet,2021

4. Diversity of CFTR variants across ancestries characterized using 454,727 UK biobank whole exome sequences;Ideozu;medRxiv,2023

5. The U.S. Food and Drug Administration's experience with ivacaftor in cystic fibrosis. Establishing efficacy using in vitro data in lieu of a clinical trial;Durmowicz;Ann Am Thorac Soc,2018

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1. Expanding the indication of CFTR modulator combinations for people with cystic fibrosis with non-F508del variants;The Lancet Respiratory Medicine;2024-08

2. The expanded French compassionate programme for elexacaftor–tezacaftor–ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study;The Lancet Respiratory Medicine;2024-08

3. Evaluation of elexacaftor–tezacaftor–ivacaftor treatment in individuals with cystic fibrosis and CFTRN1303K in the USA: a prospective, multicentre, open-label, single-arm trial;The Lancet Respiratory Medicine;2024-08

4. The Folding Pathway of ABC Transporter CFTR: Effective and Robust;Journal of Molecular Biology;2024-07

5. The clinical effectiveness of elexacaftor/tezacaftor/ivacaftor (ETI) for people with CF without a F508del variant: A systematic review and meta-analysis;Journal of Cystic Fibrosis;2024-07