GeneticsJournal of Cystic FibrosisPeer-reviewed
Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10kb C-to-T splicing mutation
Yifat S. Oren, Michal Irony‐Tur Sinai, Anita Golec…·Batsheva Kerem
This study develops an antisense oligonucleotide (ASO) therapy for CF patients with the 3849+10kb C-to-T splicing mutation, showing restoration of CFTR function in patient-derived cells.
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