Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation
针对携带 3849+10 kb C-to-T 剪接突变的囊性纤维化患者的反义寡核苷酸药物开发
期刊:Journal of Cystic Fibrosis
影响因子:5.4
doi:10.1016/j.jcf.2021.06.003
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