Upregulation of a CFTR mRNA isoform has therapeutic potential for the treatment of 3′ CFTR PTC variants

Molecular Therapy — Nucleic Acids 2026 Open access

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Authors: Norm Allaire, Matthew S. Armstrong, Jae Seok Yoon, Mercy C. Walker, Priyanka Bhatt, Jan Harrington, Yi Cheng, Eric I. Wong, John E. Mahoney, Hillary C. Valley, C. Macadino, Hermann J. Bihler
+4 more Andrey Sivachenko, Calvin U. Cotton, John P. Carulli, Martin Mense
DOI: 10.1016/j.omtn.2025.102829
Cited by: 0
References: 31

Abstract

transport function in immortalized epithelial and primary hBE cells homozygous for CFTR W1282X, respectively. This study provides a foundation for advancing ASO-mediated upregulation of e22 trunc mRNA and protein as a therapeutic approach for cystic fibrosis caused by 3'-terminal CFTR PTC mutations.

Fields of Study & Subjects

Cystic Fibrosis Research Advances Neonatal Respiratory Health Research RNA Research and Splicing

Publication details

Journal / source
Molecular Therapy — Nucleic Acids
Volume
37
Issue
1
Pages
102829–102829
Publication date
2 January 2026
Language
EN