Antisense Oligonucleotide Therapeutics for Cystic Fibrosis: Recent Developments and Perspectives.

ABSTRACT: Antisense oligonucleotide (ASO) technology has become an attractive therapeutic modality for various diseases, including Mendelian disorders. ASOs can modulate the expression of a target gene by promoting mRNA degradation or changing pre-mRNA splicing, nonsense-mediated mRNA decay, or translation. Advances in medicinal chemistry and a deeper understanding of post-transcriptional mechanisms have led to the approval of several ASO drugs for diseases that had long lacked therapeutic options. For instance, an ASO drug called nusinersen became the first approved drug for spinal muscular atrophy, improving survival and the overall disease course. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause cystic fibrosis (CF). Although Trikafta and other CFTR-mo

SUBMITTER: Kim YJ 

PROVIDER: S-EPMC9880599 | biostudies-literature | 2023 Jan

REPOSITORIES: biostudies-literature