Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT
对立体纯反义寡核苷酸进行临床前评估,以选择性降低突变型HTT的表达水平。
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2024.102246
Iwamoto, Naoki; Liu, Yuanjing; Frank-Kamenetsky, Maria; Maguire, Abbie; Tseng, Wei Chou; Taborn, Kristin; Kothari, Nayantara; Akhtar, Ali; Bowman, Keith; Shelke, Juili Dilip; Lamattina, Anthony; Hu, Xiao Shelley; Jang, Hyun Gyung; Kandasamy, Pachamuthu; Liu, Fangjun; Longo, Ken; Looby, Richard; Meena; Metterville, Jake; Pan, Qianli; Purcell-Estabrook, Erin; Shimizu, Mamoru; Prakasha, Priyanka Shiva; Standley, Stephany; Upadhyay, Hansini; Yang, Hailin; Yin, Yuan; Zhao, Anderson; Francis, Christopher; Byrne, Mike; Dale, Elena; Verdine, Gregory L; Vargeese, Chandra