CFTR functional measurements in human models for diagnosis, prognosis and personalized therapy

Author:

Beekman Jeffrey M.,Sermet-Gaudelus Isabelle,de Boeck Kris,Gonska Tanja,Derichs Nico,Mall Marcus A.,Mehta Anil,Martin Ulrich,Drumm Mitch,Amaral Margarida D.

Funder

FCT

CFF-Cystic Fibrosis Foundation, USA

Myrovlitis Trust

NCFS-Dutch CF Foundation

Wilhelmina Research

German CF Association

Christiane Herzog Foundation, Germany

Cystic Fibrosis Research Inc., USA

ECFS CTN

ECFS Diagnostic Network Working Group

German CF Association — Mukoviszidose e.V.

CF Canada

CFFT

French CF Association — Vaincre La Mucoviscidose

CFF — Cystic Fibrosis Foundation, USA

Publisher

Elsevier BV

Subject

Pulmonary and Respiratory Medicine,Pediatrics, Perinatology and Child Health

Reference69 articles.

1. CFTR function and prospects for therapy;Riordan;Annu Rev Biochem,2008

2. Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene;Sosnay;Nat Genet,2013

3. Genotype and phenotype in cystic fibrosis;Zielenski;Respiration,2000

4. CFTR biomarkers: time for promotion to surrogate endpoint?;De Boeck;Eur Respir J,2012

5. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation;Accurso;N Engl J Med,2010

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