Personalized medicine in CF: from modulator development to therapy for cystic fibrosis patients with rare CFTR mutations

Cystic fibrosis (CF) is the most common life-shortening genetic disease affecting ~1 in 3,500 of the Caucasian population. CF is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. To date, more than 2,000 CFTR mutations have been identified, which produce a wide range of...

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Bibliografiska uppgifter
I publikationen:Am J Physiol Lung Cell Mol Physiol
Huvudupphovsmän: Harutyunyan, Misak, Huang, Yunjie, Mun, Kyu-Shik, Yang, Fanmuyi, Arora, Kavisha, Naren, Anjaparavanda P.
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Physiological Society 2018
Ämnen:
Review
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC5966781/
https://ncbi.nlm.nih.gov/pubmed/29351449
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1152/ajplung.00465.2017
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