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

Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo.

利用 CRISPR-Cas9 核糖核蛋白在体内递送治疗单基因和双基因显性遗传性听力损失

Tao Yong, Lamas Veronica, Du Wan, Zhu Wenliang, Li Yiran, Whittaker Madelynn N, Zuris John A, Thompson David B, Rameshbabu Arun Prabhu, Shu Yilai, Gao Xue, Hu Johnny H, Pei Charles, Kong Wei-Jia, Liu Xuezhong, Wu Hao, Kleinstiver Benjamin P, Liu David R, Chen Zheng-Yi

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

通过体内递送基因组编辑剂治疗常染色体显性听力损失

Xue Gao, Yong Tao, Veronica Lamas, Mingqian Huang, Wei-Hsi Yeh, Bifeng Pan, Yu-Juan Hu, Johnny H Hu, David B Thompson, Yilai Shu, Yamin Li, Hongyang Wang, Shiming Yang, Qiaobing Xu, Daniel B Polley, M Charles Liberman, Wei-Jia Kong, Jeffrey R Holt, Zheng-Yi Chen, David R Liu

Aptazyme-embedded guide RNAs enable ligand-responsive genome editing and transcriptional activation

嵌入适体酶的引导RNA能够实现配体响应性基因组编辑和转录激活。

Tang, Weixin; Hu, Johnny H; Liu, David R