CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro
CRISPR/Cas9 介导切除 C9ORF72 中引起 ALS/FTD 的六核苷酸重复扩增可在体内和体外挽救主要疾病机制
期刊:Nature Communications
影响因子:14.7
doi:10.1038/s41467-022-33332-7
Katharina E Meijboom, Abbas Abdallah, Nicholas P Fordham, Hiroko Nagase, Tomás Rodriguez, Carolyn Kraus, Tania F Gendron, Gopinath Krishnan, Rustam Esanov, Nadja S Andrade, Matthew J Rybin, Melina Ramic, Zachary D Stephens, Alireza Edraki, Meghan T Blackwood, Aydan Kahriman, Nils Henninger, Jean-Pie
