Cystic Fibrosis: CFTR Correctors to the Rescue

被引：11

作者：

Sheppard, David N. ^{[1
]}

机构：

[1] Univ Bristol, Sch Physiol & Pharmacol, Bristol BS8 1TD, Avon, England

来源：

CHEMISTRY & BIOLOGY | 2011年 / 18卷 / 02期

关键词：

TRANSMEMBRANE CONDUCTANCE REGULATOR; SMALL-MOLECULE CORRECTORS; F508DEL-CFTR; TRAFFICKING; CHANNEL; MUTATION;

D O I：

10.1016/j.chembiol.2011.02.003

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

引用

页码：145 / 147

页数：3

共 12 条

[1] Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation [J].

Accurso, Frank J. ;

Rowe, Steven M. ;

Clancy, J. P. ;

Boyle, Michael P. ;

Dunitz, Jordan M. ;

Durie, Peter R. ;

Sagel, Scott D. ;

Hornick, Douglas B. ;

Konstan, Michael W. ;

Donaldson, Scott H. ;

Moss, Richard B. ;

Pilewski, Joseph M. ;

Rubenstein, Ronald C. ;

Uluer, Ahmet Z. ;

Aitken, Moira L. ;

Freedman, Steven D. ;

Rose, Lynn M. ;

Mayer-Hamblett, Nicole ;

Dong, Qunming ;

Zha, Jiuhong ;

Stone, Anne J. ;

Olson, Eric R. ;

Ordonez, Claudia L. ;

Campbell, Preston W. ;

Ashlock, Melissa A. ;

Ramsey, Bonnie W. .

NEW ENGLAND JOURNAL OF MEDICINE, 2010, 363 (21) :1991-2003

[2] A Chemical Corrector Modifies the Channel Function of F508del-CFTR [J].

Chiaw, Patrick Kim ;

Wellhauser, Leigh ;

Huan, Ling Jun ;

Ramjeesingh, Mohabir ;

Bear, Christine E. .

MOLECULAR PHARMACOLOGY, 2010, 78 (03) :411-418

[3] Gating of the CFTR Cl- channel by ATP-driven nucleotide-binding domain dimerisation [J].

Hwang, Tzyh-Chang ;

Sheppard, David N. .

JOURNAL OF PHYSIOLOGY-LONDON, 2009, 587 (10) :2151-2161

[4] Small molecule correctors of F508del-CFTR discovered by structure-based virtual screening [J].

Kalid, Ori ;

Mense, Martin ;

Fischman, Sharon ;

Shitrit, Alina ;

Bihler, Hermann ;

Ben-Zeev, Efrat ;

Schutz, Nili ;

Pedemonte, Nicoletta ;

Thomas, Philip J. ;

Bridges, Robert J. ;

Wetmore, Diana R. ;

Marantz, Yael ;

Senderowitz, Hanoch .

JOURNAL OF COMPUTER-AIDED MOLECULAR DESIGN, 2010, 24 (12) :971-991

[5] Binding site of activators of the cystic fibrosis transmembrane conductance regulator in the nucleotide binding domains [J].

Moran, O ;

Galietta, LJV ;

Zegarra-Moran, O .

CELLULAR AND MOLECULAR LIFE SCIENCES, 2005, 62 (04) :446-460

[6] Small-molecule correctors of defective ΔF508-CFTR cellular processing identified by high-throughput screening [J].

Pedemonte, N ;

Lukacs, GL ;

Du, K ;

Caci, E ;

Zegarra-Moran, O ;

Galietta, LJV ;

Verkman, AS .

JOURNAL OF CLINICAL INVESTIGATION, 2005, 115 (09) :2564-2571

[7] Solubilizing mutations used to crystallize one CFTR domain attenuate the trafficking and channel defects caused by the major cystic fibrosis mutation [J].

Pissarra, Luisa S. ;

Farinha, Carlos M. ;

Xu, Zhe ;

Schmidt, Andre ;

Thibodeau, Patrick H. ;

Cai, Zhiwei ;

Thomas, Philip J. ;

Sheppard, David N. ;

Amaral, Margaricla D. .

CHEMISTRY & BIOLOGY, 2008, 15 (01) :62-69

[8] CFTR function and prospects for therapy [J].

Riordan, John R. .

ANNUAL REVIEW OF BIOCHEMISTRY, 2008, 77 :701-726

[9] Identification of a NBD1-Binding Pharmacological Chaperone that Corrects the Trafficking Defect of F508del-CFTR [J].

Sampson, Heidi M. ;

Robert, Renaud ;

Liao, Jie ;

Matthes, Elizabeth ;

Carlile, Graeme W. ;

Hanrahan, John W. ;

Thomas, David Y. .

CHEMISTRY & BIOLOGY, 2011, 18 (02) :231-242

[10] Chimeric constructs endow the human CFTR Cl-channel with the gating behavior of murine CFTR [J].

Scott-Ward, Toby S. ;

Cai, Zhiwei ;

Dawson, Elizabeth S. ;

Doherty, Ann ;

Da Paula, Ana Carina ;

Davidson, Heather ;

Porteous, David J. ;

Wainwright, Brandon J. ;

Amaral, Margarida D. ;

Sheppard, David N. ;

Boyd, A. Christopher .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2007, 104 (41) :16365-16370

← 1 2 →