A split and inducible adenine base editor for precise in vivo base editing [PDF]
Nature Communications, 2023 DNA base editors use deaminases fused to a programmable DNA-binding protein for targeted nucleotide conversion. However, the most widely used TadA deaminases lack post-translational control in living cells.
Hongzhi Zeng +9 more
doaj +7 more sources
DNA capture by a CRISPR-Cas9-guided adenine base editor. [PDF]
Science, 2020 Secrets of a fast base editor CRISPR-Cas9 base editors comprise RNA-guided Cas proteins fused to an enzyme that can deaminate a DNA nucleoside. No natural enzyme deaminates adenine in DNA, and so a breakthrough came when a natural transfer RNA deaminase was fused to Cas9 and evolved to give an adenine base editor (
Lapinaite A +8 more
europepmc +10 more sources
Transient expression of an adenine base editor corrects the Hutchinson-Gilford progeria syndrome mutation and improves the skin phenotype in mice [PDF]
Nature Communications, 2022 Base editing to treat diseases is progressing but tissue delivery and progenitor cells correction are challenging. Here, the authors show sustained effects and propagation of mutation-corrected progenitors by transient adenine base editor expression ...
Daniel Whisenant +7 more
doaj +3 more sources
Engineering of near-PAMless adenine base editor with enhanced editing activity and reduced off-target [PDF]
Molecular Therapy: Nucleic Acids, 2022 About 47% of pathogenic point mutations could be corrected by ABE-induced A·T-to-G·C conversions. However, the applications of ABEs are still hindered by undesired editing efficiency, limited editing scopes, and off-targeting effects.
Xiaofang Cao +11 more
doaj +4 more sources
ABE8e adenine base editor precisely and efficiently corrects a recurrent COL7A1 nonsense mutation [PDF]
Scientific Reports, 2022 Base editing introduces precise single-nucleotide edits in genomic DNA and has the potential to treat genetic diseases such as the blistering skin disease recessive dystrophic epidermolysis bullosa (RDEB), which is characterized by mutations in the ...
Adam Sheriff +18 more
doaj +3 more sources
Genome-wide profiling of adenine base editor specificity by EndoV-seq [PDF]
Nature Communications, 2019 Adenine base editors are an important contribution to the genome editing toolbox. Here the authors present EndoV-seq, an endonuclease-based assay for evaluating genomewide off-target effects of base editing.
Puping Liang +11 more
doaj +5 more sources
A motif preferred adenine base editor with minimal bystander and off-targets editing [PDF]
Nature Communications47% of hereditable diseases are caused by single C•G-to-T•A base conversions, which means efficient A-to-G base editing tools (ABEs) have great potential for the treatment of these diseases. However, the existing efficient ABEs, while catalyzing targeted
Mengyu Shang +7 more
doaj +6 more sources
Efficient Gene Silencing by Adenine Base Editor-Mediated Start Codon Mutation [PDF]
Molecular Therapy, 2020 Traditional CRISPR/Cas9-based gene knockouts are generated by introducing DNA double-strand breaks (DSBs), but this may cause excessive DNA damage or cell death. CRISPR-based cytosine base editors (CBEs) and adenine base editors (ABEs) can facilitate C-to-T or A-to-G exchanges, respectively, without DSBs.
Xinjie Wang, Lu Dang, Xingxu Huang
exaly +5 more sources
Adenine base editor engineering reduces editing of bystander cytosines [PDF]
Nature Biotechnology, 2021 Adenine base editors (ABEs) catalyze specific A-to-G conversions at genomic sites of interest. However, ABEs also induce cytosine deamination at the target site. To reduce the cytosine editing activity, we engineered a commonly used adenosine deaminase, TadA7.10, and found that ABE7.10 with a D108Q mutation in TadA7.10 exhibited tenfold reduced ...
You Kyeong Jeong +7 more
core +7 more sources
A precise and efficient adenine base editor. [PDF]
Mol Ther, 2022 Adenine base editors (ABEs) are novel genome-editing tools, and their activity has been greatly enhanced by eight additional mutations, thus named ABE8e. However, elevated catalytic activity was concomitant with frequent generation of bystander mutations. This bystander effect precludes its safe applications required in human gene therapy.
Tu T +17 more
europepmc +3 more sources