Upregulation of a CFTR mRNA isoform has therapeutic potential for the treatment of 3' CFTR PTC variants
CFTR mRNA亚型的上调具有治疗3' CFTR PTC变异体的治疗潜力。
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102829
Allaire, Normand E; Armstrong, Matthew S; Yoon, Jae Seok; Walker, Mercy C; Bhatt, Priyanka; Harrington, Jan M; Cheng, Yi; Wong, Eric I; Mahoney, John E; Valley, Hillary C; Macadino, Caitlin M; Bihler, Hermann J; Sivachenko, Andrey Y; Cotton, Calvin U; Carulli, John P; Mense, Martin
