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
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 +4 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
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 +3 more sources
Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity [PDF]
Nature Biotechnology, 2022 Alvin Hsu +2 more
exaly +3 more sources
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 +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
Targeted
Plant Biotechnol J, 2023 Dating Zhong +9 more
openalex +2 more sources
Computer simulations explain mutation-induced effects on the DNA editing by adenine base editors. [PDF]
, 2020 Adenine base editors, which were developed by engineering a transfer RNA adenosine deaminase enzyme (TadA) into a DNA editing enzyme (TadA*), enable precise modification of A:T to G⋮C base pairs. Here, we use molecular dynamics simulations to uncover the
Komor, Alexis C +2 more
core +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