Sustained inhibition of ENaC in CF: Potential RNA-based therapies for mutation-agnostic treatment.

Pradeep Kota

Journal: Current opinion in pharmacology 2022;64():102209

PMID: 35483215

Full Text on Elsevier Science View on PubMed

Abstract

Disruption of the equilibrium between ion secretion and absorption processes by the airway epithelium is central to many muco-obstructive lung diseases including cystic fibrosis (CF). Besides correction of defective folding and function of CFTR, inhibition of amiloride-sensitive epithelia sodium channels (ENaC) has emerged as a bona fide therapeutic strategy to improve mucociliary clearance in patients with CF. The short half-life of amiloride-based ENaC blockers and hyperosmotic therapies have led to the development of novel RNA-based interventions for targeted and sustained reduction of ENaC expression and function in preclinical models of CF. This review summarizes the recent advances in RNA therapeutics targeting ENaC for mutation-agnostic treatment of CF.

Address: Cystic Fibrosis Research and Treatment Center, University of North Carolina at Chapel Hill, NC 27599, USA. Electronic address: [email protected].

DOI: 10.1016/j.coph.2022.102209

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MeSH Terms: Amiloride, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator, Epithelial Sodium Channel Blockers, Epithelial Sodium Channels, Humans, Mutation, RNA