Oligonucleotide-based therapies for cystic fibrosis

In the clinically successful era of CFTR modulators and Theratyping, 10–20% of individuals with cystic fibrosis (CF) may develop disease due to CFTR mutations that remain undruggable. These individuals produce low levels of CFTR mRNA and/or not enough protein to be rescued with modulator drugs. Alte...

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Bibliographic Details
Published in:Current opinion in pharmacology 2022-10, Vol.66, p.102271, Article 102271
Main Author: Kreda, Silvia M.
Format: Article
Language:English
Subjects:
Cystic Fibrosis - drug therapy
Cystic Fibrosis - genetics
Cystic Fibrosis Transmembrane Conductance Regulator - genetics
Cystic Fibrosis Transmembrane Conductance Regulator - metabolism
Humans
Mutation
Oligonucleotides - therapeutic use
Precision Medicine
RNA, Messenger
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Summary:In the clinically successful era of CFTR modulators and Theratyping, 10–20% of individuals with cystic fibrosis (CF) may develop disease due to CFTR mutations that remain undruggable. These individuals produce low levels of CFTR mRNA and/or not enough protein to be rescued with modulator drugs. Alternative therapeutic approaches to correct the CFTR defect at the mRNA level using nucleic acid technologies are currently feasible; e.g., oligonucleotides platforms, which are being rapidly developed to correct genetic disorders. Drug-like properties, great specificity, and predictable off-target effects by design make oligonucleotides a valuable approach with fewer clinical and ethical challenges than genomic editing strategies. Together with personalized and precision medicine approaches, oligonucleotides are ideal therapeutics to target CF-causing mutations that affect only a few individuals resilient to modulator therapies.
ISSN:1471-4892
1471-4973
1471-4973
DOI:10.1016/j.coph.2022.102271