Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic fibrosis.
Eluforsen: a potential new treatment option for people with cystic fibrosis (CF) due to the F508del gene mutation?
Antisense oligonucleotide targeting of mRNAs encoding ENaC subunits α, β, and ɣ improves cystic fibrosis-like disease in mice