Base editing of trinucleotide repeats that cause Huntington's disease and Friedreich's ataxia reduces somatic repeat expansions in patient cells and in mice
对导致亨廷顿病和弗里德赖希共济失调的三核苷酸重复序列进行碱基编辑，可以减少患者细胞和小鼠体内的体细胞重复序列扩增。
期刊:Nature Genetics
影响因子:31.7
doi:10.1038/s41588-025-02172-8
Zaneta Matuszek #,Mandana Arbab #,Maheswaran Kesavan,Alvin Hsu,Jennie C L Roy,Jing Zhao,Tian Yu,Ben Weisburd,Gregory A Newby,Neil J Doherty,Muzhou Wu,Shota Shibata,Ana Cristian,Y Allen Tao,Liam G Fearnley,Melanie Bahlo,Heidi L Rehm,Jun Xie,Guangping Gao,Ricardo Mouro Pinto,David R Liu
