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

Development of a DUX4-targeting antibody oligonucleotide conjugate as a therapy for FSHD

开发靶向DUX4的抗体寡核苷酸偶联物作为FSHD的治疗方法

Malecova, Barbora; Sala, David; Melikian, Garineh M; Johns, Rachel; Erdogan, Gulin; Hartmann, Marc; Jordan, Maryam; Arias, Joel Danny; Bhattacharya, Arvind; Meng, Qingying; Dansereau, Oliver; Beppler, Samuel W; Doppalapudi, Venkata R; Huang, Hanhua; Flanagan, William Michael; Levin, Arthur A

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

Novel antisense therapy targeting microRNA-132 in patients with heart failure: results of a first-in-human Phase 1b randomized, double-blind, placebo-controlled study

针对心力衰竭患者microRNA-132的新型反义疗法:一项首次人体1b期随机、双盲、安慰剂对照研究的结果

Täubel, Jörg; Hauke, Wilfried; Rump, Steffen; Viereck, Janika; Batkai, Sandor; Poetzsch, Jenny; Rode, Laura; Weigt, Henning; Genschel, Celina; Lorch, Ulrike; Theek, Carmen; Levin, Arthur A; Bauersachs, Johann; Solomon, Scott D; Thum, Thomas

Antisense-mediated reduction of proprotein convertase subtilisin/kexin type 9 (PCSK9): a first-in-human randomized, placebo-controlled trial

反义介导的降低前蛋白转化酶枯草杆菌蛋白酶/kexin 9 型 (PCSK9) 水平:一项首次人体随机、安慰剂对照试验

van Poelgeest, Eveline P; Hodges, Michael R; Moerland, Matthijs; Tessier, Yann; Levin, Arthur A; Persson, Robert; Lindholm, Marie W; Dumong Erichsen, Kamille; Ørum, Henrik; Cohen, Adam F; Burggraaf, Jacobus

Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through

恢复杜氏肌营养不良症肌肉中肌营养不良蛋白的表达,外显子跳跃和终止密码子通读的进展

Hoffman, Eric P; Bronson, Abby; Levin, Arthur A; Takeda, Shin'ichi; Yokota, Toshifumi; Baudy, Andreas R; Connor, Edward M

Inhibition of protein tyrosine phosphatase-1B with antisense oligonucleotides improves insulin sensitivity and increases adiponectin concentrations in monkeys

利用反义寡核苷酸抑制蛋白酪氨酸磷酸酶-1B可改善猴子的胰岛素敏感性并增加脂联素浓度

Swarbrick, Michael M; Havel, Peter J; Levin, Arthur A; Bremer, Andrew A; Stanhope, Kimber L; Butler, Madeline; Booten, Sheri L; Graham, James L; McKay, Robert A; Murray, Susan F; Watts, Lynnetta M; Monia, Brett P; Bhanot, Sanjay