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

Presence of phosphodiester backbone, but not nucleobases, in the guide's 3' terminal region is necessary for RISC loading and target cleavage in vitro and in vivo

引导RNA 3'末端区域中磷酸二酯骨架的存在(而非核碱基的存在)是RISC在体外和体内装载和靶标切割所必需的。

O'Reilly, Daniel; Furgal, Raymond C; Ross, Vella M; Luu, Eric; Gross, Katherine Y; Hariharan, Vignesh; Summers, Ashley; Cooper, David; Allen, Sarah; Dahlke, Christopher; Rachid, Mohamad Omar; Chan, Io Long; Fakih, Hassan H; Alterman, Julia F; Echeverria, Dimas; Watts, Jonathan K; Khvorova, Anastasia

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

Antisense oligonucleotide jacifusen for FUS-ALS: an investigator-initiated, multicentre, open-label case series

针对FUS-ALS的反义寡核苷酸jacifusen:一项研究者发起的多中心、开放标签病例系列研究

Shneider, Neil A; Harms, Matthew B; Korobeynikov, Vlad A; Rifai, Olivia M; Hoover, Benjamin N; Harrington, Elizabeth A; Aziz-Zaman, Sonya; Singleton, Jessica; Jamil, Arish; Madan, Vikram R; Lee, Ikjae; Andrews, Jinsy A; Smiley, Richard M; Alam, Mahabub M; Black, Lauren E; Shin, Minwook; Watts, Jonathan K; Walk, David; Newman, Daniel; Pascuzzi, Robert M; Weber, Markus; Neuwirth, Christoph; Da Cruz, Sandrine; Soriano, Armand; Lane, Roger; Henry, Scott; Mathews, Joel; Jafar-Nejad, Paymaan; Norris, Dan; Rigo, Frank; Brown, Robert H; Miller, Stephan; Crean, Rebecca; Bennett, C Frank

Increasing intracellular dNTP levels improves prime editing efficiency.

提高细胞内dNTP水平可提高先导编辑效率

Liu Pengpeng, Ponnienselvan Karthikeyan, Nyalile Thomas, Oikemus Sarah, Joynt Anya T, Iyer Sukanya, Kelly Karen, Guo Dongsheng, Kyawe Pyae P, Vanderleeden Emma, Redick Sambra D, Huang Lei, Chen Zexiang, Lee Jeong Min, Schiffer Celia A, Harlan David M, Wang Jennifer P, Emerson Charles P Jr, Lawson Nathan D, Watts Jonathan K, Sontheimer Erik J, Luban Jeremy, Wolfe Scot A

TDP-43-dependent mis-splicing of KCNQ2 triggers intrinsic neuronal hyperexcitability in ALS/FTD.

TDP-43 依赖的 KCNQ2 错误剪接引发 ALS/FTD 中的内在神经元过度兴奋。

Joseph Brian J, Marshall Kelly A, Harley Peter, Mann Jacob R, Alessandrini Francesco, Vanoye Carlos G, Chi Wanhao, Prudencio Mercedes, Simkin Dina, Kao Tzu-Ting, Desai Reshma R, Keuss Matthew J, Barattucci Simone, Zanovello Matteo, Mehta Puja R, DeKeyser Jean-Marc, Limone Francesco, Lee Jonathan, Brown Anna-Leigh, Leyton-Jaimes Marcel F, Nash Leslie A, Juan Irune Guerra San, Aronica Eleonora, Wainger Brian J, Shah Mala, Goswami Anand, Shneider Neil A, Dickson Dennis W, Burrone Juan, Zhang Chaolin, Wichterle Hynek, Petrucelli Leonard, Watts Jonathan K, George Alfred L Jr, Fratta Pietro, Eggan Kevin, Kiskinis Evangelos

Next generation APOBEC3 inhibitors: optimally designed for potency and nuclease stability

新一代APOBEC3抑制剂:针对效力和核酸酶稳定性进行了优化设计

Hedger, Adam K; Myint, Wazo; Lee, Jeong Min; Suchenski Loustaunau, Diego; Balachandran, Vanivilasini; Shaqra, Ala M; Kurt Yilmaz, Nese; Watts, Jonathan K; Matsuo, Hiroshi; Schiffer, Celia A

The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing

N=1协作项目:通过合作和数据共享推进定制化核酸疗法

Belgrad, Jillian; McConnell, Erin; Leonard, Stef; Nolen, Nicole; Lauffer, Marlen C; Watts, Jonathan K; Yu, Timothy; Yan, Winston X; Aartsma-Rus, Annemieke

Identifying optimal conditions for precise knock-in of exogenous DNA into the zebrafish genome.

确定将外源DNA精确敲入斑马鱼基因组的最佳条件

Oikemus Sarah, Hu Kai, Shin Masahiro, Idrizi Feston, Goodman-Khan Aliece, Kolb Amy, Ghanta Krishna S, Lee Jonathan, Wagh Atish, Wolfe Scot A, Zhu Lihua J, Watts Jonathan K, Lawson Nathan D

Albumin-binding dendrimer-conjugated siRNA enables safe and effective gene silencing throughout the central nervous system

白蛋白结合树状聚合物偶联的siRNA能够安全有效地在整个中枢神经系统中进行基因沉默。

Fakih, Hassan H; Ohara, Masahiro; Summers, Ashley; Sarli, Samantha L; Kelly, Karen; Gagnon, Rosemary; Maru, Bruktawit; Bramato, Brianna; Khvorova, Anastasia; Watts, Jonathan K

CRYPTID-exon: streamlined detection of cryptic exons from RNA-seq data

CRYPTID-exon:从RNA-seq数据中简化隐蔽外显子的检测

Khokhar, Eraj S; Brokaw, Kaitlyn; Kartje, Zachary J; Krykbaeva, Marina; Calvo-Roitberg, Ezequiel; Hedger, Adam K; Lee, Jonathan; Wagh, Atish; Watts, Jonathan K; Pai, Athma A