Systematic deletion of symmetrical CFTR exons reveals new therapeutic targets for exon skipping antisense oligonucleotides
Cecilia Peña‐Rasgado, Elvia Rodriguez-Manriquez, Miroslav Dundr, Robert J. Bridges, Michelle L. Hastings, Wren E. Michaels
This study identifies therapeutic targets for exon skipping antisense oligonucleotides by systematically analyzing CFTR isoforms generated through symmetrical exon deletion.
NAR Molecular Medicine · Oct 1, 2024Peer-reviewedMolecular Biology