Even though elexacaftor/ivacaftor/tezacaftor is effective in individuals with at least one F508del allele, thereby providing treatment for the majority of individuals with CF, there are still many CFTR mutations that are not targeted by existing CFTR modulators, including many splice site and nonsense mutations; individuals harboring two of these mutations do not have a therapy to improve the function of their CFTR. Here, CFTR is linked to cystic fibrosis.