Scientists at St. Jude Children's Research Hospital and Rockefeller University have combined their expertise to gain a better understanding of the cystic fibrosis transmembrane conductance regulator (CFTR). Mutations in CFTR ...
Mar 22, 2023
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Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells. Cold Spring Harbor Laboratory Professor Adrian Krainer leveraged ASO technology to develop the first FDA-approved treatment ...
Jul 14, 2022
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Researchers at the University of Toronto have identified hundreds of new proteins that could play a role in cystic fibrosis, and which may shed light on why some patients respond better than others to current therapies.
Feb 14, 2022
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The process of generating proteins from genes is akin to a factory, where workers follow a set of instructions that, ideally, are effective and clear. But for some people who suffer from cystic fibrosis (CF), one of their ...
Jan 27, 2022
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An experimental drug reported in Nature Communications suggests that a "path is clearly achievable" to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent ...
Jul 19, 2021
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Cystic fibrosis is caused by an inherited mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Due to this mutation, the CFTR protein doesn't embed in cell membranes to form a channel for chlorine ...
Dec 10, 2019
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A new UNC School of Medicine study shows that two cystic fibrosis (CF) drugs aimed at correcting the defected CFTR protein seem to be more effective when a patient's airway is inflamed. This is the first study to evaluate ...
Oct 11, 2018
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Researchers studying the root cause of cystic fibrosis have made a major advance in our understanding of silent gene changes with implications for the complexity of cystic fibrosis. The findings are published today in [16 ...
May 17, 2017
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Rockefeller scientists have created the first three-dimensional map of the protein responsible for cystic fibrosis, an inherited disease for which there is no cure. This achievement, described December 1 in Cell, offers the ...
Dec 1, 2016
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In experiments with isolated cystic fibrosis lung cells, University of Alabama at Birmingham researchers and colleagues from two other institutions have partially restored the lost function of those cells.
May 25, 2016
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