Atrophin-1 antisense oligonucleotide provides robust protection from pathology in a fully humanized DRPLA model.
Atrophin-1 反义寡核苷酸在完全人源化的 DRPLA 模型中提供了强大的病理保护作用。
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102815
Smith Velvet L, Gidi Bereket Z, Bragg Robert M, Cantle Jeffrey P, Ben-Varon Aliza, Noble Briana, Prades Silvia, Compton Andrea, Greenfield Julie, Korecka Joanna A, Gemos Anya, Yu Timothy, Khurana Vikram, Kordasiewicz Holly B, Zhao Hien T, Barker-Haliski Melissa, Child Daniel D, Carroll Jeffrey B