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

Systemic administration of an RNA binding and cell-penetrating antibody targets therapeutic RNA to multiple mouse models of cancer.

系统性地施用一种 RNA 结合和细胞穿透抗体,将治疗性 RNA 靶向应用于多种小鼠癌症模型

Quijano Elias, Martinez-Saucedo Diana, Ianniello Zaira, Pinto-Medici Natasha, Rackear Madison, Chen Haoting, Lola-Pereira Luiz, Liu Yanfeng, Hegan Denise, Shan Xinning, Tseng Robert, Yugawa Deanne, Chowdhury Sumedha, Khang Minsoo, Singh Jay P, Abdullah Rashed, Azhir Perisa, Kashima Soki, Woods Wendy S, Gosstola Nicholas, Turner Bruce C, Squinto Stephen, Ludwig Dale L, Bindra Ranjit S, Robert Marie E, Braun David A, Perez Pinera Pablo, Saltzman W Mark, Escobar-Hoyos Luisa F, Glazer Peter M

In vivo CRISPR base editing for treatment of Huntington's disease

利用体内 CRISPR 碱基编辑技术治疗亨廷顿病

Shirguppe, Shraddha; Gapinske, Michael; Swami, Devyani; Gosstola, Nicholas; Acharya, Pankaj; Miskalis, Angelo; Joulani, Dana; Szkwarek, Maddie G; Bhattacharjee, Abhishek; Elias, Gianna; Stilger, Michelle; Winter, Jackson; Woods, Wendy S; Anand, Daphine; Lim, Colin K W; Gaj, Thomas; Perez-Pinera, Pablo

SPLICER: A Highly Efficient Base Editing Toolbox That Enables In Vivo Therapeutic Exon Skipping

SPLICER:一种高效的碱基编辑工具箱,可实现体内治疗性外显子跳跃

Miskalis, Angelo; Shirguppe, Shraddha; Winter, Jackson; Elias, Gianna; Swami, Devyani; Nambiar, Ananthan; Stilger, Michelle; Woods, Wendy S; Gosstola, Nicholas; Gapinske, Michael; Zeballos, Alejandra; Moore, Hayden; Maslov, Sergei; Gaj, Thomas; Perez-Pinera, Pablo

Long-range cis-regulatory elements controlling GDF6 expression are essential for ear development

控制 GDF6 表达的长程顺式调控元件对耳朵发育至关重要。

Bademci, Guney; Abad, Clemer; Cengiz, Filiz B; Seyhan, Serhat; Incesulu, Armagan; Guo, Shengru; Fitoz, Suat; Atli, Emine Ikbal; Gosstola, Nicholas C; Demir, Selma; Colbert, Brett M; Seyhan, Gozde Cosar; Sineni, Claire J; Duman, Duygu; Gurkan, Hakan; Morton, Cynthia C; Dykxhoorn, Derek M; Walz, Katherina; Tekin, Mustafa