An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment.
一种靶向 CAG 扩增的非环状核酸修饰 siRNA,用于治疗多聚谷氨酰胺疾病。
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102802
Maeda Kentaro, Hirunagi Tomoki, Sahashi Kentaro, Kamiya Yukiko, Iida Madoka, Sakakibara Kenji, Onodera Kazunari, Ohyama Manabu, Okada Yohei, Okano Hideyuki, Asanuma Hiroyuki, Katsuno Masahisa