Taking into consideration stop mutations, rarer than the more common F508del, this research project aims to develop a new site-specific gene-editing technique. The researchers propose the development and use of site-specific mRNA editing through the deamination of Adenosine into Inosine, a ribonucleotide recognized as guanosine by the ribosome, which would allow the correction of stop codons in the mRNA. This new editing technique involves the use of the REPAIRv2 system and antisense oligonucleotides (ASOs) specific to the mRNA region where the stop codon is present. The researchers propose to evaluate the effectiveness of the procedure on cellular models with molecular biology techniques such as real time-PCR, western blotting, immunofluorescence and sequencing.

XIX Convention FFC Ricerca – download here a brief presentation of the project