Upregulation of a CFTR mRNA isoform has therapeutic potential for the treatment of 3′ CFTR PTC variants
Author:
Normand E. Allaire,Matthew S. Armstrong,Jae Seok Yoon,Mercy C. Walker,Priyanka Bhatt,Jan M. Harrington,Yi Cheng,Eric I. Wong,John E. Mahoney,Hillary C. Valley,Caitlin M. Macadino,Hermann J. Bihler,Andrey Y. Sivachenko,Calvin U. Cotton et al.
Publication:
Molecular Therapy - Nucleic Acids
© 2026 The Authors. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.