Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb 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, Chen Leibson, Liran Carmel, Joel Reiter, Eric J. Sorscher, Steve D. Wilton, Eitan Kerem, Steven M. Rowe, Isabelle Sermet-Gaudelus, Batsheva Kerem*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

29 Scopus citations

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Medicine & Life Sciences