Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide
强效混合骨架反义寡核苷酸抑制突变型 C9orf72 表达
期刊:Nature Medicine
影响因子:58.7
doi:10.1038/s41591-021-01557-6
Hélène Tran #, Michael P Moazami #, Huiya Yang, Diane McKenna-Yasek, Catherine L Douthwright, Courtney Pinto, Jake Metterville, Minwook Shin, Nitasha Sanil, Craig Dooley, Ajit Puri, Alexandra Weiss, Nicholas Wightman, Heather Gray-Edwards, Miklos Marosfoi, Robert M King, Thomas Kenderdine, Daniele F
信号转导
MSD
Human
Neurofilament L
