WS17.01 Novel approaches based on sequence-specific RNA editing by ADARs to correct CFTR nonsense mutations causing cystic fibrosis

Abstract

Background: It has been estimated that 10% of Cystic Fibrosis patients worldwide carry nonsense mutations that introduce a premature termination codon (PTC) in the CFTR transcript leading to the translation of a truncated and non-functional protein. Unfortunately, these patients cannot benefit from specific and approved therapies yet.