Emerging Therapeutic Approaches for Cystic Fibrosis. From Gene Editing to Personalized Medicine

Abstract excerpt

gene mutations have allowed the development of novel therapies targeting specific defects underlying CF. Some strategies are mutation specific and have already reached clinical development; some strategies include a read-through of the specific premature termination codons (read-through therapies, nonsense mediated decay pathway inhibitors for Class I mutations); correction of CFTR folding and trafficking to the...

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