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

Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

利用患者特异性体内基因编辑技术治疗罕见遗传疾病

Musunuru, Kiran; Grandinette, Sarah A; Wang, Xiao; Hudson, Taylor R; Briseno, Kevin; Berry, Anne Marie; Hacker, Julia L; Hsu, Alvin; Silverstein, Rachel A; Hille, Logan T; Ogul, Aysel N; Robinson-Garvin, Nancy A; Small, Juliana C; McCague, Sarah; Burke, Samantha M; Wright, Christina M; Bick, Sarah; Indurthi, Venkata; Sharma, Shweta; Jepperson, Michael; Vakulskas, Christopher A; Collingwood, Michael; Keogh, Katie; Jacobi, Ashley; Sturgeon, Morgan; Brommel, Christian; Schmaljohn, Ellen; Kurgan, Gavin; Osborne, Thomas; Zhang, He; Kinney, Kyle; Rettig, Garrett; Barbosa, Christopher J; Semple, Sean C; Tam, Ying K; Lutz, Cathleen; George, Lindsey A; Kleinstiver, Benjamin P; Liu, David R; Ng, Kim; Kassim, Sadik H; Giannikopoulos, Petros; Alameh, Mohamad-Gabriel; Urnov, Fyodor D; Ahrens-Nicklas, Rebecca C

Spatial profiling of gene editing by in situ sequencing in mice and macaques

利用原位测序技术对小鼠和猕猴的基因编辑进行空间分析

Janjuha, Sharan; Haenggi, Tatjana; Chamberlain, Thomas C; Rothgangl, Tanja; Kissling, Lucas; Wilhelm, Maria; Mathis, Nicolas; Boeck, Desiree; Marquart, Kim; Ioannidi, Eleonora; Moon, Woohyun J; Muramatsu, Hiromi; Vadovics, Máté; Pardi, Norbert; Semple, Sean C; Tam, Ying K; Schwank, Gerald

Systemic delivery of biotherapeutic RNA to the myocardium transiently modulates cardiac contractility in vivo.

将生物治疗性 RNA 系统性输送至心肌可暂时性地调节体内心脏收缩力

Shuvaev Vladimir V, Tam Ying K, Lee Benjamin W, Myerson Jacob W, Herbst Alan, Kiseleva Raisa Yu, Glassman Patrick M, Parhiz Hamideh, Alameh Mohamad-Gabriel, Pardi Norbert, Muramatsu Hiromi, Shuvaeva Tea I, Arguiri Evguenia, Marcos-Contreras Oscar A, Hood Elizabeth D, Brysgel Taylor V, Nong Jia, Papp Tyler E, Eaton Deborah M, Riley Rachel, Palanki Rohan, Musunuru Kiran, Brenner Jacob S, Mitchell Michael J, Ferrari Victor A, Mui Barbara L, Semple Sean C, Weppler Sherry A, Atluri Pavan, Margulies Kenneth B, Weissman Drew, Muzykantov Vladimir R

Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics

脂质纳米颗粒递送系统助力mRNA疗法的应用

Semple, Sean C; Leone, Robert; Barbosa, Christopher J; Tam, Ying K; Lin, Paulo J C