CFTR: New insights into structure and function and implications for modulation by small molecules

Bertrand Kleizen; John Hunt; Isabelle Callebaut; Tzyh-Chang Hwang; Isabelle Sermet-Gaudelus; Sylvia Hafkemeyer; David N Sheppard

doi:10.1016/j.jcf.2019.10.021

CFTR: New insights into structure and function and implications for modulation by small molecules

Bertrand Kleizen^*, John Hunt, Isabelle Callebaut, Tzyh-Chang Hwang, Isabelle Sermet-Gaudelus, Sylvia Hafkemeyer, David N Sheppard

^*Corresponding author for this work

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Abstract

Structural biology and functional studies are a powerful combination to elucidate fundamental knowledge about the cystic fibrosis transmembrane conductance regulator (CFTR). Here, we discuss the latest findings, including how clinically-approved drugs restore function to mutant CFTR, leading to better clinical outcomes for people with cystic fibrosis (CF). Despite the prospect of regulatory approval of a CFTR-targeting therapy for most CF mutations, strenuous efforts are still needed to fully comprehend CFTR structure-and-function for the development of better drugs to enable people with CF to live full and active lives.

Original language	English
Pages (from-to)	S19-S24
Number of pages	6
Journal	Journal of Cystic Fibrosis
Volume	19
Issue number	Supplement 1
Early online date	21 Nov 2019
DOIs	https://doi.org/10.1016/j.jcf.2019.10.021
Publication status	Published - Mar 2020

Keywords

membrane protein
ABC-transporter
mutations
F508del
therapy
clinical drugs
cystic fibrosis

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10.1016/j.jcf.2019.10.021

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This is the author accepted manuscript (AAM). The final published version (version of record) is available online via Elsevier at https://www.sciencedirect.com/science/article/abs/pii/S1569199319309361. Please refer to any applicable terms of use of the publisher.
Accepted author manuscript, 372 KBLicence: CC BY-NC-ND

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title = "CFTR: New insights into structure and function and implications for modulation by small molecules",

abstract = "Structural biology and functional studies are a powerful combination to elucidate fundamental knowledge about the cystic fibrosis transmembrane conductance regulator (CFTR). Here, we discuss the latest findings, including how clinically-approved drugs restore function to mutant CFTR, leading to better clinical outcomes for people with cystic fibrosis (CF). Despite the prospect of regulatory approval of a CFTR-targeting therapy for most CF mutations, strenuous efforts are still needed to fully comprehend CFTR structure-and-function for the development of better drugs to enable people with CF to live full and active lives. ",

keywords = "membrane protein, ABC-transporter, mutations, F508del, therapy, clinical drugs, cystic fibrosis",

author = "Bertrand Kleizen and John Hunt and Isabelle Callebaut and Tzyh-Chang Hwang and Isabelle Sermet-Gaudelus and Sylvia Hafkemeyer and Sheppard, {David N}",

year = "2020",

month = mar,

doi = "10.1016/j.jcf.2019.10.021",

language = "English",

volume = "19",

pages = "S19--S24",

journal = "Journal of Cystic Fibrosis",

issn = "1569-1993",

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T2 - New insights into structure and function and implications for modulation by small molecules

AU - Kleizen, Bertrand

AU - Hunt, John

AU - Callebaut, Isabelle

AU - Hwang, Tzyh-Chang

AU - Sermet-Gaudelus, Isabelle

AU - Hafkemeyer, Sylvia

AU - Sheppard, David N

PY - 2020/3

Y1 - 2020/3

N2 - Structural biology and functional studies are a powerful combination to elucidate fundamental knowledge about the cystic fibrosis transmembrane conductance regulator (CFTR). Here, we discuss the latest findings, including how clinically-approved drugs restore function to mutant CFTR, leading to better clinical outcomes for people with cystic fibrosis (CF). Despite the prospect of regulatory approval of a CFTR-targeting therapy for most CF mutations, strenuous efforts are still needed to fully comprehend CFTR structure-and-function for the development of better drugs to enable people with CF to live full and active lives.

AB - Structural biology and functional studies are a powerful combination to elucidate fundamental knowledge about the cystic fibrosis transmembrane conductance regulator (CFTR). Here, we discuss the latest findings, including how clinically-approved drugs restore function to mutant CFTR, leading to better clinical outcomes for people with cystic fibrosis (CF). Despite the prospect of regulatory approval of a CFTR-targeting therapy for most CF mutations, strenuous efforts are still needed to fully comprehend CFTR structure-and-function for the development of better drugs to enable people with CF to live full and active lives.

KW - membrane protein

KW - ABC-transporter

KW - mutations

KW - F508del

KW - therapy

KW - clinical drugs

KW - cystic fibrosis

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U2 - 10.1016/j.jcf.2019.10.021

DO - 10.1016/j.jcf.2019.10.021

M3 - Article (Academic Journal)

C2 - 31759907

SN - 1569-1993

VL - 19

SP - S19-S24

JO - Journal of Cystic Fibrosis

JF - Journal of Cystic Fibrosis

IS - Supplement 1

ER -

CFTR: New insights into structure and function and implications for modulation by small molecules

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CFTR: New insights into structure and function and implications for modulation by small molecules

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