Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation.
Oren YS, Irony-Tur Sinai M, Golec A, Barchad-Avitzur O, Mutyam V, Li Y, Hong J, Ozeri-Galai E, Hatton A, Leibson C, Carmel L, Reiter J, Sorscher EJ, Wilton SD, Kerem E, Rowe SM, Sermet-Gaudelus I, Kerem B.
Oren YS, et al. Among authors: irony tur sinai m.
J Cyst Fibros. 2021 Sep;20(5):865-875. doi: 10.1016/j.jcf.2021.06.003. Epub 2021 Jul 2.
J Cyst Fibros. 2021.
PMID: 34226157
Free PMC article.