CFTR and cystic fibrosis: However, further evaluation through clinical trials is warranted [62]. The promising exploration of CFTR modulators has significantly benefited the majority of CF patients, addressing gating and trafficking defects of the mutated CFTR protein; however, around 10% of patients remain unresponsive to these modulators due to specific mutations or intolerances, making gene therapy a mutation-independent and attractive option for this group, mainly targeting life-threatening lung disease until gene therapy advances to treat multiple affected organs in CF [63].