Wild-type, stabilized and CF-causing CFTR variants are available ... “The allosteric effects of the F508del mutation in human CFTR elucidated by cryo-EM enabled by protein engineering,” presented by ...
provided across widely used durable cell types (e.g., FRT, Calu-3) and airway-specific cell expression systems emerging in their importance (e.g., 16HBE14o-, CFBE41o-), are essential for therapeutic ...
CF is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, resulting in abnormal chloride ...
However, more extensive studies of CFTR mutations in larger and more diverse populations have shown that mutations in additional genes could perhaps modulate the severity and type of disease ...
Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR ...
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The long breath: Medical advancements giving cystic fibrosis patients a fuller lifeThe development of other types of antibiotics during ... More than 2,500 CF-causing mutations have been discovered since then ...
New study suggests why mice with cystic fibrosis mutations can stave off bacterial infections in ... The genetic defect that causes cystic fibrosis cripples these defenses. CFTR proteins normally ...
Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR ...
An additional 94 non-F508del CFTR mutations and a boxed warning on liver injury and liver failure were included in the updated prescribing information. 1507_1515del9, 2183A→G, A1067P ...
ALYFTREK also offers a potentially transformative option for approximately 150 people with CF in the U.S. with one of 31 mutations who are now eligible for a CFTR modulator for the first time.
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