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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A team of researchers from UCLA, Cedars-Sinai and the Cystic Fibrosis Foundation has developed a first-of-its-kind molecular catalog of cells in healthy lungs and the lungs of people with cystic fibrosis.
May 6, 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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If you have two faulty copies of the CFTR gene, you will have cystic fibrosis. But the severity of your disease will depend on environmental factors and many other genes. Locating these genes and altering their function could ...
Sep 29, 2015
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(Medical Xpress)—Cystic fibrosis is caused by a mutation in the gene that encodes a particular protein, known as the cystic fibrosis transmembrane conductance regulator (or CFTR). Although this discovery was made 25 years ...
Jul 9, 2013
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Researchers at the University of Iowa Carver College of Medicine have discovered a genetic process that can restore function to a defective protein, which is the most common cause of cystic fibrosis (CF).
Aug 1, 2012
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Two nationally recognized experts in cloning and stem cell science from the University of Houston, Wa Xian and Frank McKeon, are reporting that five lung stem cell variants dominate the lungs of patients with advanced cystic ...
Sep 27, 2023
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Cystic fibrosis is a rare genetic disease which can cause very serious symptoms. In particular, patients suffer from chronic bacterial infections that can lead to respiratory failure. It is caused by mutations in the CFTR ...
May 24, 2022
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An international, open-label Phase 3 study, co-led by Susanna McColley, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago, found that a regimen of three drugs (elexacaftor/tezacaftor/ivacaftor) that targets the ...
Mar 18, 2021
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