Allele-specific targeting of mutant ataxin-3 by antisense oligonucleotides in SCA3-iPSC-derived neurons
反义寡核苷酸在 SCA3-iPSC 衍生的神经元中对突变型 Ataxin-3 进行等位基因特异性靶向
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.5
doi:10.1016/j.omtn.2021.11.015
Stefan Hauser, Jacob Helm, Melanie Kraft, Milena Korneck, Jeannette Hübener-Schmid, Ludger Schöls
