A pathogenic proteolysis-resistant huntingtin isoform induced by an antisense oligonucleotide maintains huntingtin function
由反义寡核苷酸诱导产生的具有致病性、抗蛋白水解能力的亨廷顿蛋白亚型可维持亨廷顿蛋白的功能。
期刊:JCI Insight
影响因子:6.1
doi:10.1172/jci.insight.154108
Kim, Hyeongju; Lenoir, Sophie; Helfricht, Angela; Jung, Taeyang; Karneva, Zhana K; Lee, Yejin; Beumer, Wouter; van der Horst, Geert B; Anthonijsz, Herma; Buil, Levi Cm; van der Ham, Frits; Platenburg, Gerard J; Purhonen, Pasi; Hebert, Hans; Humbert, Sandrine; Saudou, Frédéric; Klein, Pontus; Song, Ji-Joon
