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

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

Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression

在脱唾液酸糖蛋白受体表达降低的临床前动物模型中评估 GalNAc-siRNA 结合活性

Jennifer L S Willoughby, Amy Chan, Alfica Sehgal, James S Butler, Jayaprakash K Nair, Tim Racie, Svetlana Shulga-Morskaya, Tuyen Nguyen, Kun Qian, Kristina Yucius, Klaus Charisse, Theo J C van Berkel, Muthiah Manoharan, Kallanthottathil G Rajeev, Martin A Maier, Vasant Jadhav, Tracy S Zimmermann

Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification

使用循环 RNA 定量技术进行皮下 ALAS1 RNAi 治疗肝卟啉症的临床前开发

Amy Chan, Abigail Liebow, Makiko Yasuda, Lin Gan, Tim Racie, Martin Maier, Satya Kuchimanchi, Don Foster, Stuart Milstein, Klaus Charisse, Alfica Sehgal, Muthiah Manoharan, Rachel Meyers, Kevin Fitzgerald, Amy Simon, Robert J Desnick, William Querbes