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 +5 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
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
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 +2 more sources
Structure-guided engineering of adenine base editor with minimized RNA off-targeting activity [PDF]
Nature Communications, 2021 Base editors can induce transcriptome-wide RNA off-target editing independent of gRNA. Here, the authors engineer ABEmax variants with minimized RNA off-target activities.
Jianan Li +8 more
doaj +2 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 +3 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 +2 more sources
Protein-nucleic acid language model-assisted design of precise and compact adenine base editor [PDF]
Nature CommunicationsAdenine base editors (ABEs) are powerful tools for gene therapy. However, efficient version of ABEs (e.g. ABE8e) always induce excessive bystander and off-target editing events and are large in size, hindering their potential in clinical disease ...
Jingxuan Ren +10 more
doaj +2 more sources
Adenine base editor–mediated splicing remodeling activates noncanonical splice sites [PDF]
Journal of Biological Chemistry, 2023 Qing Li
exaly +2 more sources
Engineering an adenine base editor in human embryonic stem cells with minimal DNA and RNA off-target activities [PDF]
Molecular Therapy - Nucleic Acids, 2022 Xingxu Huang +2 more
exaly +2 more sources