Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT

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

Molecular therapy. Nucleic acids, 2024-09, Vol.35 (3), p.102246, Article 102246 [Periódico revisado por pares]

Elsevier Inc

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