Therapeutic base editing of human hematopoietic stem cells

J Zeng, Y Wu, C Ren, J Bonanno, AH Shen, D Shea… - Nature Medicine, 2020 - nature.com
… (rAPO1) in the BE3 with an engineered human APOBEC3A (A3A) domain 13 . The N57Q …
context within the base editing window. We recovered this base editor protein with high …

[HTML][HTML] Development of a simple and quick method to assess base editing in human cells

X Lv, K Qiu, T Tu, X He, Y Peng, J Ye, J Fu… - … Therapy-Nucleic Acids, 2020 - cell.com
… for quick detection of editing activity and specificity of base editors (BEs) in humaneditors).
Our system could be harnessed as a base editing assessment platform, which would pave …

[HTML][HTML] In vivo somatic cell base editing and prime editing

GA Newby, DR Liu - Molecular Therapy, 2021 - cell.com
base editors and prime editors can serve as effective agents for in vivo therapeutic genome
editingcell (post-natal) base editing and prime editing and prospects for future development. …

[HTML][HTML] Base editing in human cells with monomeric DddA-TALE fusion deaminases

YG Mok, JM Lee, E Chung, J Lee, K Lim, SI Cho… - Nature …, 2022 - nature.com
… ) can be fused to dCas9 or nCas9 to create novel base editors with altered editing windows.
These base editors can be used for base editing at positions upstream or downstream of a …

Base editing the mammalian genome

EM Schatoff, MP Zafra, LE Dow - Methods, 2019 - Elsevier
… DNA base editing, and discuss the recent progress toward the development of optimized base
editing systems for mammalian cells. … examples of successful base editing in cell lines and …

[HTML][HTML] Therapeutic adenine base editing of human hematopoietic stem cells

J Liao, S Chen, S Hsiao, Y Jiang, Y Yang… - Nature …, 2023 - nature.com
… for editing human CD34 + HSPCs while the guide RNA-dependent DNA off-target editing is
greatly reduced when base editor … on-target and off-target base editing by either the ABE8e + …

Base editing: precision chemistry on the genome and transcriptome of living cells

HA Rees, DR Liu - Nature reviews genetics, 2018 - nature.com
base editors achieve analogous changes using components that target RNA. Base editors
directly convert one base or base … -dividing cells without generating excess undesired editing

[HTML][HTML] Targeted A-to-G base editing in human mitochondrial DNA with programmable deaminases

SI Cho, S Lee, YG Mok, K Lim, J Lee, JM Lee, E Chung… - Cell, 2022 - cell.com
… In an effort to create adenine base editors enabling A-to-G substitutions in organelle DNA, …
bind to the ND1 or ND4 gene in human mitochondria, a deoxyadenosine deaminase variant, …

Highly efficient base editing in human tripronuclear zygotes

C Zhou, M Zhang, Y Wei, Y Sun, Y Sun, H Pan… - … & cell, 2017 - academic.oup.com
… We carried out base editing in human 3PN zygotes by microinjection of BE3 mRNA and …
4- to 8-cell embryos with FANCF or DNMT3B base editing at the single-cell level. Single …

[HTML][HTML] Correction of the Marfan syndrome pathogenic FBN1 mutation by base editing in human cells and heterozygous embryos

Y Zeng, J Li, G Li, S Huang, W Yu, Y Zhang, D Chen… - Molecular therapy, 2018 - cell.com
… In this study, taking advantage of the BE3 base editing system, we achieved specific correction
of a MFS pathogenic FBN1 mutation first in a human cell line and then in human zygotic …