CNS-targeted base editing of the major late-onset Tay-Sachs mutation alleviates disease in mice.
针对中枢神经系统的主要迟发性泰-萨克斯病突变进行碱基编辑可缓解小鼠的疾病
期刊:Journal of Clinical Investigation
影响因子:13.6
doi:10.1172/JCI183434
Allende Maria L, Kono Mari, Lee Y Terry, Olmsted Samantha M, Huso Vienna, Bakir Jenna Y, Pratto Florencia, Li Cuiling, Byrnes Colleen, Tuymetova Galina, Zhu Hongling, Tifft Cynthia J, Proia Richard L