Antisense Oligonucleotide Therapeutics for Cystic Fibrosis: Recent Developments and Perspectives

Kim, Young Jin, Krainer, Adrian R (January 2023) Antisense Oligonucleotide Therapeutics for Cystic Fibrosis: Recent Developments and Perspectives. Molecules and Cells, 46 (1). pp. 10-20. ISSN 1016-8478

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DOI: 10.14348/molcells.2023.2172


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-modulation therapies benefit most CF patients, there is a significant unmet therapeutic need for a subset of CF patients. In this review, we introduce ASO therapies and their mechanisms of action, describe the opportunities and challenges for ASO therapeutics for CF, and discuss the current state and prospects of ASO therapies for CF.

Item Type: Paper
Subjects: therapies
diseases & disorders > congenital hereditary genetic diseases > cystic fibrosis
bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > oligonucleotide
CSHL Authors:
Communities: CSHL labs > Krainer lab
SWORD Depositor: CSHL Elements
Depositing User: CSHL Elements
Date: 31 January 2023
Date Deposited: 06 Feb 2023 17:25
Last Modified: 06 Feb 2023 17:25

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