Toward inclusive therapy with CFTR modulators: Progress and challenges

Author:

Guimbellot Jennifer12ORCID,Sharma Jyoti23,Rowe Steven M.1234ORCID

Affiliation:

1. Departments of Pediatrics; The University of Alabama at Birmingham; Birmingham Alabama

2. Gregory Fleming James Cystic Fibrosis Research Center; The University of Alabama at Birmingham; Birmingham Alabama

3. Cell Developmental and Integrative Biology; The University of Alabama at Birmingham; Birmingham Alabama

4. Departments of Medicine; The University of Alabama at Birmingham; Birmingham Alabama

Publisher

Wiley

Subject

Pulmonary and Respiratory Medicine,Pediatrics, Perinatology and Child Health

Reference139 articles.

1. Genetic medicines for CF: hype versus reality;Alton;Pediatr Pulmonol,2016

2. Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease;Lee;Cochrane Database Syst Rev,2016

3. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation;Ramsey;N Engl J Med,2011

4. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770;Van Goor;Proc Natl Acad Sci USA,2009

5. Clinical mechanism of the CFTR potentiator ivacaftor in G551D-Mediated cystic fibrosis;Rowe;Am J Respir Crit Care Med,2014

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