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

In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene

通过暂时抑制必需基因实现基因靶向肝细胞的体内扩增

Marco De Giorgi, So Hyun Park, Adam Castoreno, Mingming Cao, Ayrea Hurley, Lavanya Saxena, Marcel A Chuecos, Christopher J Walkey, Alexandria M Doerfler, Mia N Furgurson, M Cecilia Ljungberg, Kalyani R Patel, Sarah Hyde, Tyler Chickering, Stephanie Lefebvre, Kelly Wassarman, Patrick Miller, June Qin

From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization

从实验室到临床:利用种子配对不稳定提高 GalNAc-siRNA 结合物的临床安全性

Mark K Schlegel, Maja M Janas, Yongfeng Jiang, Joseph D Barry, Wendell Davis, Saket Agarwal, Daniel Berman, Christopher R Brown, Adam Castoreno, Sarah LeBlanc, Abigail Liebow, Tara Mayo, Stuart Milstein, Tuyen Nguyen, Svetlana Shulga-Morskaya, Sarah Hyde, Sally Schofield, John Szeto, Lauren Blair Wo

Overcoming GNA/RNA base-pairing limitations using isonucleotides improves the pharmacodynamic activity of ESC+ GalNAc-siRNAs

使用同核苷酸克服 GNA/RNA 碱基配对限制可提高 ESC+ GalNAc-siRNA 的药效学活性

Mark K Schlegel, Shigeo Matsuda, Christopher R Brown, Joel M Harp, Joseph D Barry, Daniel Berman, Adam Castoreno, Sally Schofield, John Szeto, Muthiah Manoharan, Klaus Charissé, Martin Egli, Martin A Maier

Small circular interfering RNAs (sciRNAs) as a potent therapeutic platform for gene-silencing

小环状干扰 RNA(sciRNA)作为基因沉默的有效治疗平台

Hartmut Jahns, Rohan Degaonkar, Peter Podbevsek, Swati Gupta, Anna Bisbe, Krishna Aluri, John Szeto, Pawan Kumar, Sarah LeBlanc, Tim Racie, Christopher R Brown, Adam Castoreno, Dale C Guenther, Vasant Jadhav, Martin A Maier, Janez Plavec, Martin Egli, Muthiah Manoharan, Ivan Zlatev

Chimeric siRNAs with chemically modified pentofuranose and hexopyranose nucleotides: altritol-nucleotide (ANA) containing GalNAc-siRNA conjugates: in vitro and in vivo RNAi activity and resistance to 5'-exonuclease

具有化学修饰的戊呋喃糖和己吡喃糖核苷酸的嵌合 siRNA:含有 GalNAc-siRNA 结合物的阿卓糖醇核苷酸 (ANA):体外和体内 RNAi 活性和对 5'-外切酶的抗性

Pawan Kumar, Rohan Degaonkar, Dale C Guenther, Mikhail Abramov, Guy Schepers, Marie Capobianco, Yongfeng Jiang, Joel Harp, Charalambos Kaittanis, Maja M Janas, Adam Castoreno, Ivan Zlatev, Mark K Schlegel, Piet Herdewijn, Martin Egli, Muthiah Manoharan

An investigational RNAi therapeutic targeting Factor XII (ALN-F12) for the treatment of hereditary angioedema

一项针对因子 XII (ALN-F12) 的在研 RNAi 疗法,用于治疗遗传性血管性水肿

Jingxuan Liu, June Qin, Anna Borodovsky, Timothy Racie, Adam Castoreno, Mark Schlegel, Martin A Maier, Tracy Zimmerman, Kevin Fitzgerald, James Butler, Akin Akinc

Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

选择具有有限脱靶驱动大鼠肝毒性的 GalNAc 结合 siRNA

Maja M Janas, Mark K Schlegel, Carole E Harbison, Vedat O Yilmaz, Yongfeng Jiang, Rubina Parmar, Ivan Zlatev, Adam Castoreno, Huilei Xu, Svetlana Shulga-Morskaya, Kallanthottathil G Rajeev, Muthiah Manoharan, Natalie D Keirstead, Martin A Maier, Vasant Jadhav