Cytosine and adenine base editing of the brain, liver, retina, heart ...
2020年1月14日 · Optimized dual AAVs enable in vivo base editing at therapeutically relevant efficiencies and dosages in the mouse brain (up to 59% of unsorted cortical tissue), liver …
Screening an Effective Dual-Adeno-Associated Virus Split
Adeno-associated virus (AAV) has been widely used for in vivo gene therapy, but its limited 4.7 kb packing capacity presents challenges in delivering CBE by a single AAV. To address this, one …
In vivo base editing restores sensory transduction and ... - AAAS
To repair the mutation, we tested several optimized cytosine base editors (CBEmax variants) and guide RNAs in Baringo mouse embryonic fibroblasts. We packaged the most promising CBE, …
Detect-seq reveals out-of-protospacer editing and target ... - Nature
2021年6月7日 · Here we report Detect-seq for the evaluation of CBE specificity. It enables sensitive detection of CBE-induced off-target sites at the genome-wide level. Detect-seq …
Efficient prime editing in mouse brain, liver and heart with
2023年5月4日 · We apply these systems to install putative protective mutations in vivo for Alzheimer’s disease in astrocytes and for coronary artery disease in hepatocytes. In vivo prime …
Treatment of a Mouse Model of ALS by In Vivo Base Editing
2020年4月8日 · In this study, we establish an intein-based trans -splicing system that enables the in vivo delivery of cytidine base editors (CBEs) consisting of the Streptococcus pyogenes …
In vivo base editing restores sensory transduction and transiently ...
2020年6月3日 · To repair the mutation, we tested several optimized cytosine base editors (CBEmax variants) and guide RNAs in Baringo mouse embryonic fibroblasts. We packaged …
In vivo cytidine base editing of hepatocytes without detectable off ...
Supporting this hypothesis, we found that ex vivo in HEK293T cells RNA off-target effects were critically dependent on excessive CBE overexpression. Notably, analysis of genome wide off …
A New‐Generation Base Editor with an Expanded Editing Window …
2024年4月11日 · The excellent editing performance of the dBhCas12b-based CBE and dBhCas12b-based ABE can efficiently produce a mutant library with much greater diversity in …
江南大学周哲敏教授团队独立创制出微生物 “新一代宽编辑窗口碱 …
2024年4月16日 · 近日，Advanced Science（IF=15.1）在线发表了江南大学生物工程学院周哲敏教授团队的研究成果“A New-Generation Base Editor with An Expanded Editing Window for …