Make a short story long: About novel long-non coding RNA regulator of CFTR gene expression

Cystic fibrosis (CF) is a genetic disorder caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) gene. Introduction of highly effective modulator therapy (HEMT) targeted at some of the CFTR protein defects was a major breakthrough in the treatment of people with cystic fibrosis. This therapy is effective in carriers of at least one allele of CFTR delF508 mutation; however, patients carrying other mutations are not eligible for this therapy.1 Moreover, some delF508 carriers are intolerant to the HEMT therapy; therefore, there is still a large group of patients that does not have any therapeutic opportunity to choose from.