Suppression of CFTR premature termination codons and rescue of CFTR protein and function by the synthetic aminoglycoside NB54

Parallel effects of VX-770 on transepithelial potential difference in vitro and in vivo

(2010) S.M. Rowe et al.   Journal of Cystic Fibrosis

ΔF508 CFTR processing correction and activity in polarized airway and non-airway cell monolayers

(2010) S.M. Rowe et al.   PULMONARY PHARMACOLOGY & THERAPEUTICS

Activation of the Cystic Fibrosis Transmembrane Conductance Regulator by the Flavonoid Quercetin

(2009) Louise C. Pyle et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Pharmaceuticals Targeting Nonsense Mutations in Genetic Diseases

(2009) Steven M. Rowe et al.   BIODRUGS

Activation of Intestinal Cl− Secretion by Lubiprostone Requires the Cystic Fibrosis Transmembrane Conductance Regulator

(2009) Marcel J.C. Bijvelds et al.   GASTROENTEROLOGY

Poly-l-aspartic Acid Enhances and Prolongs Gentamicin-mediated Suppression of theCFTR-G542X Mutation in a Cystic Fibrosis Mouse Model

(2009) Ming Du et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Development of Novel Aminoglycoside (NB54) with Reduced Toxicity and Enhanced Suppression of Disease-Causing Premature Stop Mutations

(2009) Igor Nudelman et al.   JOURNAL OF MEDICINAL CHEMISTRY

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

(2009) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial

(2008) Eitan Kerem et al.   LANCET

PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model

(2008) M. Du et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA