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, Ofra Barchad-Avitzur, Venkateshwar Mutyam, Li Yao, Jeong Shin Hong, Efrat Ozeri-Galai, Aurélie Hatton, Chen Leibson, Liran Carmel, Joel M. Reiter, Eric J. Sorscher, Steve Donald Wilton, Eitan Kerem, , 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.
Journal of Cystic Fibrosis · Jul 2, 2021Peer-reviewedGenetics