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

Acyclic serinol nucleic acid modification of siRNAs overcomes seed region mediated off-target effects while maintaining potency

siRNA的非环状丝氨醇核酸修饰可克服种子区介导的脱靶效应,同时保持效力。

Chickering, Tyler; Harp, Joel M; Jiang, Yongfeng; Qin, June; He, Guo; Hyde, Sarah; Ihlefeld, Audrey; Janas, Maja; O'Flaherty, Derek K; Schofield, Sally; Szeto, John; Theile, Christopher S; Charissé, Klaus; Jadhav, Vasant; Maier, Martin A; Manoharan, Muthiah; Egli, Martin; Schlegel, Mark K

Identifying colorectal cancer-specific vulnerabilities in the Wnt-driven long non-coding transcriptome.

识别 Wnt 驱动的长非编码转录组中结直肠癌特异性脆弱性

Schwarzmueller Laura J, Adam Ronja S, Moreno Leandro F, Nijman Lisanne E, Logiantara Adrian, Eleonora Steven, Bril Oscar, Vromans Sophie, de Groot Nina E, Giugliano Francesca Paola, Stepanova Ekaterina, Muncan Vanesa, Elbers Clara C, Lenos Kristiaan J, Zwijnenburg Danny A, van Eijndhoven Monique A J, Pegtel Dirk Michiel, van Neerven Sanne M, Loayza-Puch Fabricio, Dadali Tulin, Broom Wendy J, Maier Martin A, Koster Jan, Vermeulen Louis, Léveillé Nicolas

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

Role of a "Magic" Methyl: 2'-Deoxy-2'-α-F-2'-β-C-methyl Pyrimidine Nucleotides Modulate RNA Interference Activity through Synergy with 5'-Phosphate Mimics and Mitigation of Off-Target Effects

“神奇”甲基的作用:2'-脱氧-2'-α-F-2'-β-C-甲基嘧啶核苷酸通过与5'-磷酸类似物协同作用并减轻脱靶效应来调节RNA干扰活性。

Guenther, Dale C; Mori, Shohei; Matsuda, Shigeo; Gilbert, Jason A; Willoughby, Jennifer L S; Hyde, Sarah; Bisbe, Anna; Jiang, Yongfeng; Agarwal, Saket; Madaoui, Mimouna; Janas, Maja M; Charisse, Klaus; Maier, Martin A; Egli, Martin; Manoharan, Muthiah

Biodegradable lipids enabling rapidly eliminated lipid nanoparticles for systemic delivery of RNAi therapeutics

可生物降解的脂质能够使脂质纳米颗粒快速清除,从而实现RNAi疗法的全身递送。

Maier, Martin A; Jayaraman, Muthusamy; Matsuda, Shigeo; Liu, Ju; Barros, Scott; Querbes, William; Tam, Ying K; Ansell, Steven M; Kumar, Varun; Qin, June; Zhang, Xuemei; Wang, Qianfan; Panesar, Sue; Hutabarat, Renta; Carioto, Mary; Hettinger, Julia; Kandasamy, Pachamuthu; Butler, David; Rajeev, Kallanthottathil G; Pang, Bo; Charisse, Klaus; Fitzgerald, Kevin; Mui, Barbara L; Du, Xinyao; Cullis, Pieter; Madden, Thomas D; Hope, Michael J; Manoharan, Muthiah; Akinc, Akin

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

聚乙二醇脂质解吸速率对siRNA脂质纳米颗粒药代动力学和药效学的影响

Mui, Barbara L; Tam, Ying K; Jayaraman, Muthusamy; Ansell, Steven M; Du, Xinyao; Tam, Yuen Yi C; Lin, Paulo Jc; Chen, Sam; Narayanannair, Jayaprakash K; Rajeev, Kallanthottathil G; Manoharan, Muthiah; Akinc, Akin; Maier, Martin A; Cullis, Pieter; Madden, Thomas D; Hope, Michael J

Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo

最大程度提高siRNA脂质纳米颗粒在体内肝脏基因沉默中的效力

Jayaraman, Muthusamy; Ansell, Steven M; Mui, Barbara L; Tam, Ying K; Chen, Jianxin; Du, Xinyao; Butler, David; Eltepu, Laxman; Matsuda, Shigeo; Narayanannair, Jayaprakash K; Rajeev, Kallanthottathil G; Hafez, Ismail M; Akinc, Akin; Maier, Martin A; Tracy, Mark A; Cullis, Pieter R; Madden, Thomas D; Manoharan, Muthiah; Hope, Michael J

Unique gene-silencing and structural properties of 2'-fluoro-modified siRNAs

2'-氟修饰siRNA独特的基因沉默和结构特性

Manoharan, Muthiah; Akinc, Akin; Pandey, Rajendra K; Qin, June; Hadwiger, Philipp; John, Matthias; Mills, Kathy; Charisse, Klaus; Maier, Martin A; Nechev, Lubomir; Greene, Emily M; Pallan, Pradeep S; Rozners, Eriks; Rajeev, Kallanthottathil G; Egli, Martin

Peptide nucleic acids conjugated to short basic peptides show improved pharmacokinetics and antisense activity in adipose tissue

与短碱性肽偶联的肽核酸在脂肪组织中表现出改善的药代动力学和反义活性。

Wancewicz, Edward V; Maier, Martin A; Siwkowski, Andrew M; Albertshofer, Klaus; Winger, Theodore M; Berdeja, Andres; Gaus, Hans; Vickers, Timothy A; Bennett, C Frank; Monia, Brett P; Griffey, Richard H; Nulf, Christopher J; Hu, Jiaxin; Corey, David R; Swayze, Eric E; Kinberger, Garth A