[Treatment perspectives for patients not eligible for therapies for cystic fibrosis].

TREATMENT PERSPECTIVES FOR PATIENTS NOT ELIGIBLE FOR PROTEIN THERAPIES FOR CYSTIC FIBROSIS. The vast majority of patients with cystic fibrosis can benefit from proteic therapy. 10% of patients remain without therapeutic access. RNA editing strategies such as antisense oligonucleotides for specific splice mutations or RNA editing are now in the clinical phase. Gene therapy, DNA editing or readthrough therapies face numerous challenges in terms of vectorization and efficiency. Strong interactions between academic laboratories, harmaceutical industries, associations and health authorities may be unable to consider that in the near future, cystic fibrosis is no longer a f atal disease.