Beyond seek and destroy: How to generate allelic series using genome editing tools [PDF]
, 2020 Genome editing tools have greatly facilitated the functional analysis of genes of interest by targeted mutagenesis. Many usable genome editing tools, including different site-specific nucleases and editor databases that allow single-nucleotide ...
Bes, Martine +10 more
core +3 more sources
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
Sequence Instability in the Proviral Long Terminal Repeat and gag Regions from Peripheral Blood and Tissue-Derived Leukocytes of FIV-Infected Cats during the Late Asymptomatic Phase. [PDF]
, 2016 Feline immunodeficiency virus (FIV) infection results in viral persistence, a prolonged asymptomatic phase, and progressive immunopathology. During the asymptomatic phase, a cohort of experimentally FIV-infected cats exhibits features of viral latency in
Eckstrand, Christina D +2 more
core +3 more sources
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
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
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
Cofactor Selectivity in Methylmalonyl Coenzyme A Mutase, a Model Cobamide-Dependent Enzyme. [PDF]
, 2019 Cobamides, a uniquely diverse family of enzyme cofactors related to vitamin B12, are produced exclusively by bacteria and archaea but used in all domains of life.
Mok, Kenny +5 more
core +1 more source
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