A cystic fibrosis cure for all? Gene editing shows promise
There's been advancement on cystic fibrosis, but there are still those living with the disease who get no relief from current treatments. Gene editing may help.
www.nhlbi.nih.govI can share a quick update on CFTR news, but I don’t have live browsing access here. If you’d like, I can summarize recent themes and point you to reliable sources to check now.
What CFTR refers to: CFTR is the gene encoding the cystic fibrosis transmembrane conductance regulator protein, whose dysfunction causes cystic fibrosis (CF) and also has roles studied in cancer and other diseases. Recent public discussion often centers on modulators that improve CFTR protein function or expression, and on novel approaches like gene editing or molecular prosthetics. For context, approved CFTR modulators have substantially changed CF management in many patients, though access and mutation-specific efficacy remain important issues [general CFTR context].
Recent themes you’ll see in current news:
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There's been advancement on cystic fibrosis, but there are still those living with the disease who get no relief from current treatments. Gene editing may help.
www.nhlbi.nih.govCLEVELAND - Results of the pivotal Phase 3 clinical trial published in the Nov. 3, 2011 New England Journal of Medicine, find that the oral medicatio...
case.eduLARVOL VERI predictive biomarker news, CFTR (CF Transmembrane Conductance Regulator)
veri.larvol.comCystic fibrosis lung cells were restored to 50 percent of healthy function in work that provides the first evidence that novel therapeutic strategies for human patients can be identified based on yeast studies.
www.uab.eduA new study confirms ivacaftor's impact on inflammation in people with cystic fibrosis (CF). Ivacaftor targets the defective protein that causes CF.
news.cuanschutz.eduHow are a football match, a plug socket and a birthday cake linked to CFTR, the gene that causes cystic fibrosis? Find out by reading our ‘Research blast’ article celebrating 30 years since the gene was discovered.
www.cysticfibrosis.org.ukProfessor Tzyh-Chang Hwang Deciphers Pathogenic Protein Structure, Advancing Drug Development for Cystic Fibrosis and Diarrheal Diseases
www.nycu.edu.twA Potential Breakthrough Treatment for Cystic Fibrosis Enters Clinical Trial Led by CI MED and U of Iowa Researchers
medicine.illinois.edua collaboration with the CF Foundation (CFF). This CF research program would work to find medications, called modulators, to correct defective CFTR protein. Since the start of this collaboration, Vertex has developed Kalydeko® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and … paved the way for other CFTR modulators that may benefit many more patients. In 2015, lumacaftor/ivacaftor (Orkambi) combination therapy was approved by the FDA for patients with two F508del mutations (the most common CF...
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