日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy.

AOC 1044 可诱导外显子 44 跳跃,并在杜氏肌营养不良症的临床前模型中恢复肌营养不良蛋白

Etxaniz Usue, Marks Isaac, Albin Tyler, Diaz Matthew, Bhardwaj Raghav, Anderson Aaron, Tyaglo Olecya, Hoang Tiffany, Missinato Maria Azzurra, Svensson Kristoffer, Badillo Ben, Kovach Philip R, Leung Laura, Cochran Michael, Kwon Hae Won, Ahad Shah Md Nur, Maruyama Rika, Yokota Toshifumi, Doppalapudi Venkata R, Darimont Beatrice, Younis Husam S, Flanagan W Michael, Levin Arthur A, Huang Hanhua, Karamanlidis Georgios

Targeted tissue delivery of RNA therapeutics using antibody-oligonucleotide conjugates (AOCs)

利用抗体-寡核苷酸偶联物(AOCs)进行RNA疗法的靶向组织递送

Malecova, Barbora; Burke, Rob S; Cochran, Michael; Hood, Michael D; Johns, Rachel; Kovach, Philip R; Doppalapudi, Venkata R; Erdogan, Gulin; Arias, J Danny; Darimont, Beatrice; Miller, Christopher D; Huang, Hanhua; Geall, Andrew; Younis, Husam S; Levin, Arthur A