Correction of mutations within the cystic fibrosis transmembrane conductance regulator by site-directed RNA editing

RNA editing by adenosine deamination is a natural process of site-directed mutagenesis used by organisms to modify genetic information as it passes through RNA. In this paper we present an engineered RNA editing enzyme that can be induced to edit any adenosine that is chosen. We show that our system can efficiently correct a premature stop codon in the cystic fibrosis transmembrane conductance regulator in frog oocytes. Furthermore, a fully genetically encoded version of the system functions in human cells. As a general method, site-directed RNA editing could be a useful technique for correcting genetic mutations and modifying protein function.