نتایج جستجو برای: r117h

تعداد نتایج: 90  

Journal: :The American journal of physiology 1999
J P Clancy F E Ruiz E J Sorscher

ATP and its metabolites stimulate Cl- secretion in human epithelium in vitro and in vivo. The specific purinergic receptor subtypes that govern these effects have been difficult to separate, in part due to multiple parallel pathways for Cl- secretion in respiratory and intestinal epithelia. In a simplified model using COS-7 cells, we demonstrate acquisition of an ATP-, ADP-, AMP-, and adenosine...

2016
S. Guigui J. Wang R.I. Cohen

INTRODUCTION Ivacaftor, a cystic fibrosis transmembrane regulator (CFTR) potentiator is currently approved for use in individuals with class III gating mutations and the R117H mutation, a non-gating mutation with residual functioning CFTR. Nevertheless, ivacaftor may also be effective in individuals who have CF mutations giving rise to a residual functioning protein. However, aside from case re...

Journal: :American journal of physiology. Lung cellular and molecular physiology 2016
Mark J Turner Elizabeth Matthes Arnaud Billet Amy J Ferguson David Y Thomas Scott H Randell Lawrence E Ostrowski Kathy Abbott-Banner John W Hanrahan

Cystic fibrosis (CF), a genetic disease caused by mutations in the CFTR gene, is a life-limiting disease characterized by chronic bacterial airway infection and severe inflammation. Some CFTR mutants have reduced responsiveness to cAMP/PKA signaling; hence, pharmacological agents that elevate intracellular cAMP are potentially useful for the treatment of CF. By inhibiting cAMP breakdown, phosph...

Journal: :American journal of respiratory and critical care medicine 2000
P G Noone C A Pue Z Zhou K J Friedman E L Wakeling M Ganeshananthan R H Simon L M Silverman M R Knowles

Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) gene. The 5T allele in intron 8 (IVS8) causes abnormal splicing in the CFTR gene, and is associated with lung disease when it occurs in cis with a missense mutation in the CFTR gene, R117H. However, the 5T variant alone has not been reported to cause lung disease. We describe two adult female patients w...

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