A Combination Therapy for Cystic Fibrosis
نویسندگان
چکیده
The most prevalent form of cystic fibrosis arises from an amino acid deletion in the cystic fibrosis transmembrane conductance regulator, CFTR. A recently approved treatment for individuals homozygous for this mutation combines a chemical corrector, which helps CFTR fold, and a potentiator that increases CFTR channel activity.
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ورودعنوان ژورنال:
- Cell
دوره 163 شماره
صفحات -
تاریخ انتشار 2015