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    #ECFS2018 — Can Remote Monitoring Really Revolutionize Cystic Fibrosis Care?

    Monday, June 18, 2018 -- Some 318,000 health-related software apps are now on the market, with another 200 appearing every day. Yet a British cystic fibrosis expert questions how effective they are at managing lung disease, for reasons that range from the needless anxiety they can cause to sporadic use and concerns with patient data protection. Jane Davies, MD, a pediatric […] The post #ECFS2018 — Can Remote Monitoring Really Revolutionize Cystic Fibrosis Care? appeared first on Cystic Fibrosis News Today.

    A G542X cystic fibrosis mouse model for examining nonsense mutation directed therapies

    Wednesday, June 20, 2018 -- by Daniel R. McHugh, Miarasa S. Steele, Dana M. Valerio, Alexander Miron, Rachel J. Mann, David F. LePage, Ronald A. Conlon, Calvin U. Cotton, Mitchell L. Drumm, Craig A. Hodges Nonsense mutations are present in 10% of patients with CF, produce a premature termination codon in CFTR mRNA causing early termination of translation, and lead to lack of CFTR function. There are no currently available animal models which contain a nonsense mutation in the endogenous Cftr locus that can be utilized to test nonsense mutation therapies. In this study, we create a CF mouse model carrying the G542X nonsense mutation in Cftr using CRISPR/Cas9 gene editing. The G542X mouse model has reduced Cftr mRNA levels, demonstrates absence of CFTR

    Vertex doubles size of cystic fibrosis drug discovery site

    Tuesday, June 19, 2018 -- Vertex has more than doubled the size of its San Diego research site that discovered key cystic fibrosis drugs. The newly expanded site covers 170,000 square feet, boosting Vertex’s ability to hunt for the next generation of blockbusters.