Cystic fibrosis: therapies targeting specific gene defects.

Abstract excerpt

Cystic Fibrosis (CF) is caused by a large number of mutations in the CFTR gene, leading to specific classes of protein defects. This review discusses these classes, an understanding of which has paved the way for novel treatment strategies. The progress in this field, through from basic research to, in one case, application for license, is described.

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