Article
Strategies for the etiological therapy of cystic fibrosis.
Cell death and differentiation - 1 Nov 2017
Maiuri Luigi, Raia Valeria, Kroemer Guido
Abstract excerpt
Etiological therapies aim at repairing the underlying cause of cystic fibrosis (CF), which is the functional defect of the cystic fibrosis transmembrane conductance regulator (CFTR) protein owing to mutations in the CFTR gene. Among these, the F508del CFTR mutation accounts for more than two thirds of CF cases worldwide. Two somehow antinomic schools of thought conceive CFTR repair in a different manner....
Topics
- Clinical Trials as Topic
- Cystic Fibrosis
- Cystic Fibrosis Transmembrane Conductance Regulator
- Humans
- Mutation
- Precision Medicine