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, Yao Li, Jeong Hong, Efrat Ozeri-Galai, Aurélie Hatton, Joel Reiter, Eric J. Sorscher, Steve D. Wilton, Eitan Kerem, Steven M. Rowe, Isabelle Sermet-Gaudelus, Batsheva Kerem
bioRxiv 2021.02.14.431123; doi: https://doi.org/10.1101/2021.02.14.431123