Results 41 to 50 of about 19,262 (234)
In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice
Nature Communications, 2022 Hemochromatosis is a metabolic disorder caused by mutations in the HFE gene. Here, the authors show that a single administration of AAV8 vectors expressing an Adenine Base Editor facilitates efficient in vivo gene correction in hepatocytes and leads to ...
Alice Rovai +13 more
doaj +1 more source
Development of plant cytosine base editors with the Cas12a system
Crop Journal, 2023 Base editors of the Cas9 system have been widely used for precise nucleotide substitution in crops. In this study, Cas12a was applied to construct plant cytosine base editors (CBEs).
Huanhuan Wang +15 more
doaj +1 more source
Communications Biology, 2021 Jiao Fan, Yige Ding, et al. examine the impact of cytosine and adenine deaminases on transcriptome-wide gene expression and splicing in HeLa and HEK293T cells.
Jiao Fan +9 more
doaj +1 more source
Sequence-specific prediction of the efficiencies of adenine and cytosine base editors
Nature Biotechnology, 2020 Base editors, including adenine base editors (ABEs)1 and cytosine base editors (CBEs)2,3, are widely used to induce point mutations. However, determining whether a specific nucleotide in its genomic context can be edited requires time-consuming experiments.
Myungjae Song +14 more
openaire +3 more sources
In vivo adenine base editing corrects newborn murine model of Hurler syndrome
Molecular Biomedicine, 2023 Mucopolysaccharidosis type I (MPS I) is a severe disease caused by loss-of-function mutation variants in the α-L-iduronidase (Idua) gene. In vivo genome editing represents a promising strategy to correct Idua mutations, and has the potential to ...
Jing Su +10 more
doaj +1 more source
Molecular Therapy: Nucleic Acids, 2021 Base editors are RNA-guided deaminases that enable site-specific nucleotide transitions. The targeting scope of these Cas-deaminase fusion proteins critically depends on the availability of a protospacer adjacent motif (PAM) at the target locus and is ...
Lukas Villiger +5 more
doaj +1 more source
TadA orthologs enable both cytosine and adenine editing of base editors
Nature Communications, 2023 AbstractCytidine and adenosine deaminases are required for cytosine and adenine editing of base editors respectively, and no single deaminase could enable concurrent and comparable cytosine and adenine editing. Additionally, distinct properties of cytidine and adenosine deaminases lead to various types of off-target effects, including Cas9-indendepent ...
Shuqian Zhang +9 more
openaire +3 more sources
Expanded base editing in rice and wheat using a Cas9-adenosine deaminase fusion
Genome Biology, 2018 Nucleotide base editors in plants have been limited to conversion of cytosine to thymine. Here, we describe a new plant adenine base editor based on an evolved tRNA adenosine deaminase fused to the nickase CRISPR/Cas9, enabling A•T to G•C conversion at ...
Chao Li +7 more
doaj +1 more source
Targeted exon skipping with AAV-mediated split adenine base editors [PDF]
Cell Discovery, 2019 AbstractTechniques for exclusion of exons from mature transcripts have been applied as gene therapies for treating many different diseases. Since exon skipping has been traditionally accomplished using technologies that have a transient effect, it is particularly important to develop new techniques that enable permanent exon skipping.
Winter, Jackson +7 more
openaire +2 more sources
ACBE, a new base editor for simultaneous C-to-T and A-to-G substitutions in mammalian systems
BMC Biology, 2020 Background Many favorable traits of crops and livestock and human genetic diseases arise from multiple single nucleotide polymorphisms or multiple point mutations with heterogeneous base substitutions at the same locus.
Jingke Xie +20 more
doaj +1 more source