
Human cell based directed evolution of adenine base editors …
2021年10月8日 · Adenine base editors (ABE) are genome-editing tools that have been harnessed to introduce precise A•T to G•C conversion. However, the low activity of ABE at...
A precise and efficient adenine base editor - ScienceDirect
2022年9月7日 · NG-ABE9e exhibits robust and precise base-editing activity in human cells, with more than 7-fold bystander editing reduction at some sites, compared with NG-ABE8e. To demonstrate its practical utility, we used NG-ABE9e to correct the frequent T17M mutation in Rhodopsin for autosomal dominant retinitis pigmentosa.
Engineering a precise adenine base editor with minimal bystander ...
2022年10月13日 · Adenine base editors (ABEs) catalyze A-to-G transitions showing broad applications, but their bystander mutations and off-target editing effects raise safety concerns.
Efficient generation of mouse models of human diseases via ABE …
2018年6月14日 · Here we achieve A-to-G conversion in vivo at frequencies up to 100% by microinjection of ABE mRNA together with sgRNAs. We then generate mouse models harboring clinically relevant mutations at Ar...
大幅提高安全性,谷峰/张金伟/高彩霞合作开发精准高效碱基编辑器|编辑|碱基|编辑器|NG-ABE…
2022年8月2日 · 碱基编辑器 (Base Editor)通过基因组 DNA 中的单核苷酸转换实现精确的基因校正,而无需产生DSB,从而克服了 CRISPR 系统的许多限制,然而 脱靶效应 和 旁观者效应 却阻碍了碱基编辑器在人类基因治疗中的安全应用。 近日,温州医科大学 谷峰 课题组、美国国立卫生研究院 张金伟 、中国科学院遗传与发育生物学研究所 高彩霞 合作在 Molecular Therapy 期刊发表了题为:A precise and efficient adenine base editor 的研究论文。 该研究开发了 更精确的下 …
A precise and efficient adenine base editor - PubMed
2022年9月7日 · We identify a novel variant (NG-ABE9e), which harbors nine mutations. NG-ABE9e exhibits robust and precise base-editing activity in human cells, with more than 7-fold bystander editing reduction at some sites, compared with NG-ABE8e.
Adenine base editor-mediated splicing remodeling activates
Adenine base editors (ABEs) are genome-editing tools that have been harnessed to introduce precise A•T to G•C conversion. The discovery of split genes revealed that all introns contain two highly conserved dinucleotides, canonical "AG" (acceptor) and …
Human cell based directed evolution of adenine base editors with ...
2021年10月8日 · Adenine base editors (ABE) are genome-editing tools that have been harnessed to introduce precise A•T to G•C conversion. However, the low activity of ABE at certain sites remains a major bottleneck that precludes efficacious applications. Here, to address it, we develop a directional screening syste …
Small-molecule compounds boost genome-editing efficiency of …
2021年7月30日 · We reported a high-throughput human cells-based reporter system that can be harnessed for quickly measuring editing activity of CBE. Screening of 1813 small-molecule compounds resulted in the identification of Ricolinostat (an HDAC6 inhibitor) that can enhance the efficiency of BE3 in human cells (2.45- to 9.21-fold improvement).
A precise and efficient adenine base editor - Cell Press
2022年9月7日 · To reduce the frequent bystander mutations of NG-ABE8e, Gu and his colleagues identified a novel ABE variant (NG-ABE9e) through combinatorial engineering. NG-ABE9e exhibits robust and precise base-editing activity in human cells and rice. This study provides a versatile and improved adenine base editor for genome editing.