Antisense Oligonucleotide Therapeutics for Cystic Fibrosis: Recent Developments and Perspectives
Young Jin Kim, Adrian R. Krainer
Abstract
) 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.
Topics & Concepts
Cystic fibrosis transmembrane conductance regulatorCystic fibrosisMedicineRNA splicingGenetic enhancementBioinformaticsAntisense therapySpinal muscular atrophyIvacaftorCancer researchTranslation (biology)DiseaseOligonucleotidePharmacologyGeneMessenger RNABiologyPathologyInternal medicineRNAGeneticsLocked nucleic acidCystic Fibrosis Research AdvancesNeurogenetic and Muscular Disorders ResearchAdvanced biosensing and bioanalysis techniques