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Correction of MYBPC3 c.772G>A Splice-Site Mutation by Base Editing Ameliorates Hypertrophic Cardiomyopathy in Humanized Mice. [PDF]

Circ Res
Zhang Y, Jiao S, Yang G, Wang J, Yang X, Li Z.   +5 more
europepmc   +1 more source

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Heterologous Expression and Purification of a CRISPR-Cas9-Based Adenine Base Editor

Methods in Molecular Biology, 2023
CRISPR-cas9-guided adenine base editors (ABEs) site-specifically convert the A-T base pair to G-C base pair in genomic DNA. The intracellular delivery of ABE proteins preassembled with guide RNAs (gRNAs) has shown greatly reduced off-target effects compared with that of plasmids or viral vectors containing ABE and gRNA-encoding sequences. For efficient
Seu-Na Lee, Jae-Sung Woo
exaly   +3 more sources

Dual base editor catalyzes both cytosine and adenine base conversions in human cells

Nature Biotechnology, 2020
Although base editors are useful tools for precise genome editing, current base editors can only convert either adenines or cytosines. We developed a dual adenine and cytosine base editor (A&C-BEmax) by fusing both deaminases with a Cas9 nickase to achieve C-to-T and A-to-G conversions at the same target site. Compared to single base editors, A&C-BEmax'
Biyun Zhu, Shuming Yin, Honghui Han
exaly   +3 more sources

Adenine base editors catalyze cytosine conversions in human cells

Nature Biotechnology, 2019
Adenine base editors comprise an adenosine deaminase, evolved in vitro, and a Cas9 nickase. Here, we show that in addition to converting adenine to guanine, adenine base editors also convert cytosine to guanine or thymine in a narrow editing window (positions 5-7) and in a confined TC*N sequence context.
Heon Seok Kim, You Kyeong Jeong, Junho K Hur, Jin-Soo Kim, Sangsu Bae   +4 more
openaire   +2 more sources

Genome Engineering Human ESCs or iPSCs with Cytosine and Adenine Base Editors

2022
The ability to engineer specific mutations in human embryonic stem cells (ECSs) or induced pluripotent stem cells (iPSCs) is extremely important in the modeling of human diseases and the study of biological processes. While CRISPR/Cas9 can robustly generate gene knockouts (KOs) and gene loci modifications in coding sequences of iPSCs, it remains ...
Giulia, Pavani, Joshua G, Klein, Deborah L, French, Paul, Gadue   +3 more
openaire   +2 more sources

An adenine base editor variant expands context compatibility

Nature Biotechnology
Adenine base editors (ABEs) are precise gene-editing agents that convert A:T pairs into G:C through a deoxyinosine intermediate. Existing ABEs function most effectively when the target A is in a TA context. Here we evolve the Escherichia coli transfer RNA-specific adenosine deaminase (TadA) to generate TadA8r, which extends potent deoxyadenosine ...
Yu-Lan Xiao, Yuan Wu, Weixin Tang
openaire   +2 more sources

Germline Editing of Drosophila Using CRISPR-Cas9-Based Cytosine and Adenine Base Editors

The CRISPR Journal, 2023
Target-AID, BE3, and ABE7.10 base editors fused to the catalytically modified Cas9 and xCas9(3.7) were tested for germline editing of the fruit fly Drosophila melanogaster . We developed a guide RNA-expressing construct, white- 4gRNA, targeting splice ...
Nirav Thakkar, Adela Hejzlarova, Vaclav Brabec, David Dolezel   +3 more
openaire   +3 more sources

Hypercompact adenine base editors based on transposase B guided by engineered RNA.

Nature chemical biology, 2022
Transposon-associated transposase B (TnpB) is deemed an ancestral protein for type V, Cas12 family members, and the closest ancestor to UnCas12f1. Previously, we reported a set of engineered guide RNAs supporting high indel efficiency for Cas12f1 in human cells.
Do Yon, Kim, Yuhee, Chung, Yujin, Lee, Dongmin, Jeong, Kwang-Hyun, Park, Hyun Jung, Chin, Jeong Mi, Lee, Seyeon, Park, Sumin, Ko, Jeong-Heon, Ko, Yong-Sam, Kim   +10 more
openaire   +2 more sources

Precision genome editing using cytosine and adenine base editors in mammalian cells

Nature Protocols, 2021
Genome editing has transformed the life sciences and has exciting prospects for use in treating genetic diseases. Our laboratory developed base editing to enable precise and efficient genome editing while minimizing undesired byproducts and toxicity associated with double-stranded DNA breaks.
Tony P. Huang, Gregory A. Newby, David R. Liu   +2 more
openaire   +2 more sources

An AI-designed adenine base editor

Abstract Adenine base editors (ABEs) allow the efficient programmable conversion of adenine to guanine without causing DNA double strand breaks. Previous ABEs were generated by multiple rounds of directed evolution or derived by rational design based on the evolved ones.
Ye Yuan, Yang Chen, Rui Liu, Gula Que, Yina Yuan, Guipeng Li   +5 more
openaire   +1 more source