Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia
新型 AAV 介导的基因组编辑疗法可改善甲基丙二酸血症小鼠模型的健康和生存
期刊:PLoS One
影响因子:2.9
doi:10.1371/journal.pone.0274774
Shengwen Zhang, Amy Bastille, Susana Gordo, Nikhil Ramesh, Jenisha Vora, Elizabeth McCarthy, Xiaohan Zhang, Dylan Frank, Chih-Wei Ko, Carmen Wu, Noel Walsh, Shreya Amarwani, Jing Liao, Qiang Xiong, Lauren Drouin, Matthias Hebben, Kyle Chiang, B Nelson Chau