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

An Aptamer-Based EXACT Anticoagulant as a Sustainable, Animal-Free Alternative to Unfractionated Heparin

一种基于适配体的EXACT抗凝剂,可作为普通肝素的可持续、无动物成分替代品。

Yu, Haixiang; Pitoc, George; Zhang, Manqi; Clancy, Jeffrey; Frederiksen, James; Yan, Amy; Francis, Samuel; Sullenger, Rebecca; Rempel, Rachel; Gammell, Susannah; Caiazzi, Jillian; Sullenger, Bruce A

An siRNA targeting S6k1 identifies photoreceptor phospholipid metabolism as a contributor to lipid buildup in age-related macular degeneration.

针对 S6k1 的 siRNA 发现光感受器磷脂代谢是导致年龄相关性黄斑变性中脂质积累的因素之一。

Cheng Shun-Yun, Giguere Delaney, Kim San, Seddon Johanna M, Caiazzi Jillian, Gross Katherine, McHugh Nicholas, Echeverria Dimas, Alterman Julia F, Gray-Edwards Heather, Benatti Hector Ribeiro, Renner Lauren, Woolard Hannah, Stoddard Jonathan, McGill Trevor J, Neuringer Martha, Brush Richard S, Agbaga Martin-Paul, Khvorova Anastasia, Punzo Claudio

Organyl 5'-Phosphates in siRNA Guide Strands: Structure-Function Relationships Governing Anchoring in Argonaute 2 and Metabolic Stability

siRNA引导链中的有机5'-磷酸酯:调控Argonaute 2锚定和代谢稳定性的结构-功能关系

Carrigan-Broda, Theodore; Gebert, Luca F R; Hildebrand, Samuel; Yamada, Nozomi; Luu, Eric; Caiazzi, Jillian; McHugh, Nicholas; Echeverria, Dimas; Wagh, Atish; Watts, Jonathan K; Khvorova, Anastasia; MacRae, Ian J; Yamada, Ken

Potent and durable gene modulation in heart and muscle with chemically defined lipophilic siRNAs

利用化学成分明确的亲脂性siRNA在心脏和肌肉中实现高效持久的基因调控

Fakih, Hassan H; Lochmann, Clemens; Gagnon, Rosemary; Summers, Ashley; Caiazzi, Jillian; Buchwald, Julianna E; Tang, Qi; Maru, Bruktawit; Hildebrand, Samuel R; Abideen, Mohammad Zain U I; Furgal, Raymond C; Gross, Katherine Y; Yang, Yeon-Suk; Cooper, David; Monopoli, Kathryn R; Echeverria, Dimas; Shim, Jae-Hyuck; Yamada, Ken; Alterman, Julia F; Khvorova, Anastasia

Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model

单次注射靶向 MECP2 的治疗性二价 siRNA 可使 MECP2 重复小鼠模型免于死亡一年。

Hariharan, Vignesh N; Summers, Ashley; Clipperton-Allen, Amy E; Caiazzi, Jillian; Hildebrand, Samuel R; O'Reilly, Daniel; Tang, Qi; Kennedy, Zachary; Echeverria, Dimas; McHugh, Nicholas; Cooper, David; Souza, Jacqueline; Ferguson, Chantal; Bogdanik, Laurent; Coenraads, Monica; Khvorova, Anastasia

Phosphatidylcholine head group chemistry alters the extrahepatic accumulation of lipid-conjugated siRNA

磷脂酰胆碱头部基团的化学性质会改变脂质偶联siRNA在肝外的积累。

Hariharan, Vignesh N; Nakamura, Takahiro; Shin, Minwook; Tang, Qi; Sontakke, Vyankat; Caiazzi, Jillian; Hildebrand, Samuel; Khvorova, Anastasia; Yamada, Ken

Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors

单次玻璃体内注射四价 siRNA 可在小鼠和猪的光感受器中表现出强大而有效的基因沉默

Shun-Yun Cheng, Jillian Caiazzi, Annabelle Biscans, Julia F Alterman, Dimas Echeverria, Nicholas McHugh, Matthew Hassler, Samson Jolly, Delaney Giguere, Joris Cipi, Anastasia Khvorova, Claudio Punzo

PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs

PK 修饰锚定物可显著改变 siRNA 治疗的清除动力学、组织分布和疗效

Bruno M D C Godinho, Emily G Knox, Samuel Hildebrand, James W Gilbert, Dimas Echeverria, Zachary Kennedy, Reka A Haraszti, Chantal M Ferguson, Andrew H Coles, Annabelle Biscans, Jillian Caiazzi, Julia F Alterman, Matthew R Hassler, Anastasia Khvorova

Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity

结构受限的膦酸酯核苷酸间键影响寡核苷酸-酶相互作用,并调节 siRNA 活性和等位基因特异性

Ken Yamada, Samuel Hildebrand, Sarah M Davis, Rachael Miller, Faith Conroy, Ellen Sapp, Jillian Caiazzi, Julia F Alterman, Loic Roux, Dimas Echeverria, Matthew R Hassler, Edith L Pfister, Marian DiFiglia, Neil Aronin, Anastasia Khvorova

Docosanoic acid conjugation to siRNA enables functional and safe delivery to skeletal and cardiac muscles.

将二十二碳酸与 siRNA 结合,可实现功能性且安全的递送至骨骼肌和心肌

Biscans Annabelle, Caiazzi Jillian, McHugh Nicholas, Hariharan Vignesh, Muhuri Manish, Khvorova Anastasia