CFTR and cystic fibrosis: Cystic fibrosis is a life-limiting autosomal-recessive disease caused by mutations in the CFTR gene, while CFTR-mRNA transfection markedly restores impaired CFTR function in vitro.427 Nasally administered LNPs-CFTR mRNA was reported to result in recovery of up to 55% of the net chloride efflux characteristic in healthy mice.428 Furthermore, MRT5005, as an mRNA-based CFTR protein, has entered phase I/II clinical research.148