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

RNA editing for the treatment of alpha-1 antitrypsin deficiency.

RNA 编辑用于治疗 α-1 抗胰蛋白酶缺乏症。

Monian Prashant, Shivalila Chikdu, Lu Genliang, Bowman Keith, Briem Stearne, Bylsma Marissa, Byrne Michael, Cannon Megan, Chatterjee Arindom, Dallaire Morgan, Desai Jigar, Dufresne Jason, Faraone Alyse, Favaloro Frank, Friend Stephen, Ghosh Anamitra, Godfrey Jack, Hernandez Nidia, Huth Olivia, Iwamoto Naoki, Kawamoto Tomomi, Lamattina Anthony, Lemaitre Muriel, Lindsey Amber, Liu Fangjun, Looby Richard, Luu Khoa, McGlynn Andrew, Metterville Jake, Paik Ik-Hyeon, Pan Qianli, Patil Saurabh, Pu Tom, Purcell-Estabrook Erin, Ranade Ashwini, Rheinhardt Jeanette, Rossi Jeff, Shimizu Mamoru, Singh Kuldeep, Standley Stephany, Thomas Carina, Tripathi Snehlata, Yang Hailin, Yordanoff Ryan, Yin Yuan, Yu Hui, Narayanan PadmaKumar, Kandasamy Pachamuthu, Giangrande Paloma H, Vargeese Chandra

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。

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

Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species

立体化学增强了Malat1反义寡核苷酸在体外和体内多种物种中的效力、功效和持久性

Byrne, Michael; Vathipadiekal, Vinod; Apponi, Luciano; Iwamoto, Naoki; Kandasamy, Pachamuthu; Longo, Kenneth; Liu, Fangjun; Looby, Richard; Norwood, Lauren; Shah, Anee; Shelke, Juili Dilip; Shivalila, Chikdu; Yang, Hailin; Yin, Yuan; Guo, Lankai; Bowman, Keith; Vargeese, Chandra

Molecular imaging of oxidized collagen quantifies pulmonary and hepatic fibrogenesis

氧化胶原蛋白的分子成像可量化肺和肝纤维化的发生情况。

Chen, Howard H; Waghorn, Philip A; Wei, Lan; Tapias, Luis F; Schühle, Daniel T; Rotile, Nicholas J; Jones, Chloe M; Looby, Richard J; Zhao, Gaofeng; Elliott, Justin M; Probst, Clemens K; Mino-Kenudson, Mari; Lauwers, Gregory Y; Tager, Andrew M; Tanabe, Kenneth K; Lanuti, Michael; Fuchs, Bryan C; Caravan, Peter

In vivo molecular imaging of thrombosis and thrombolysis using a fibrin-binding positron emission tomographic probe

利用纤维蛋白结合正电子发射断层扫描探针进行血栓形成和溶栓的体内分子成像

Ay, Ilknur; Blasi, Francesco; Rietz, Tyson A; Rotile, Nicholas J; Kura, Sreekanth; Brownell, Anna Liisa; Day, Helen; Oliveira, Bruno L; Looby, Richard J; Caravan, Peter