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

Modified unlocked nucleic acid (MUNA) mitigates off-target effects of small interfering RNAs

修饰型未靶向核酸(MUNA)可减轻小干扰RNA的脱靶效应

Mori, Shohei; Datta, Dhrubajyoti; Perkins, Lydia; Jung, Michelle; Woods, Lauren Blair; Eaton, Alex; Qin, June; Racie, Tim; Kim, MaryBeth; Guenther, Dale C; Castoreno, Adam; Schlegel, Mark K; Charisse, Klaus; Egli, Martin; Matsuda, Shigeo; Manoharan, Muthiah

High resistance barrier and prophylactic protection in preclinical models of SARS-CoV-2 with two siRNA combination.

两种 siRNA 组合在 SARS-CoV-2 临床前模型中表现出高抵抗屏障和预防性保护作用

Anglero-Rodriguez Yesseinia I, Lempp Florian A, Subramanian Megha, McIninch James, Schlegel Mark K, Bohan Dana, Wong Emily, Brown Christopher R, Foster Donald J, Castoreno Adam B, Nguyen Tuyen, Cuffe Dara, Montiel-Ruiz Martin, Kaiser Hannah, Sahakyan Anna, Spreafico Roberto, Morskaya Svetlana Shulga, Barry Joseph D, Berman Daniel, Zhang Ligang, Lefebvre Stephanie, Kasper Anne, Racie Timothy, Weddle Diann, Mobley Melissa, Wassarman Kelly, Bisbe Anna, Zlatev Ivan, Rogers Arlin, Nechev Lubomir, Dybowski Joseph, Chong Saeho, Nair Jayaprakash, Simon Amy, Sloan Kevin, Hwang Seungmin, Virgin Herbert W, Fitzgerald Kevin, Maier Martin A, Hinkle Gregory, Hebner Christy M, Akinc Akin, Jadhav Vasant

Improving the potency prediction for chemically modified siRNAs through insights from molecular modeling of individual sequence positions

通过对单个序列位置的分子建模分析,提高化学修饰siRNA效力预测的准确性

Kliuchnikov, Evgenii; Maksudov, Farkhad; Zuber, Jeffrey; Hyde, Sarah; Castoreno, Adam; Waldron, Scott; Schlegel, Mark K; Marx, Kenneth A; Maier, Martin A; Barsegov, Valeri

RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes

RNAi 介导的变阻器用于动态控制 AAV 传递的转基因

Megha Subramanian, James McIninch, Ivan Zlatev, Mark K Schlegel, Charalambos Kaittanis, Tuyen Nguyen, Saket Agarwal, Timothy Racie, Martha Arbaiza Alvarado, Kelly Wassarman, Thomas S Collins, Tyler Chickering, Christopher R Brown, Karyn Schmidt, Adam B Castoreno, Svetlana Shulga-Morskaya, Elena Stam

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

LDL-Cholesterol Reduction by ANGPTL3 Inhibition in Mice Is Dependent on Endothelial Lipase

抑制 ANGPTL3 可降低小鼠体内的低密度脂蛋白胆固醇,而这种作用依赖于内皮脂肪酶

Liya Wu, Mangala M Soundarapandian, Adam B Castoreno, John S Millar, Daniel J Rader