Results 61 to 70 of about 19,445 (257)

Replacing the SpCas9 HNH domain by deaminases generates compact base editors with an alternative targeting scope

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, Lukas Schmidheini, Nicolas Mathis, Tanja Rothgangl, Kim Marquart, Gerald Schwank   +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, Luu, Alan, Gapinske, Michael, Manandhar, Sony, Shirguppe, Shraddha, Woods, Wendy S., Song, Jun S., Perez-Pinera, Pablo   +7 more
openaire   +2 more sources

Analysis and minimization of cellular RNA editing by DNA adenine base editors [PDF]

Science Advances, 2019
We report cellular RNA editing by adenine base editors and new ABE variants with reduced RNA editing activity.
Holly A. Rees, Christopher Wilson, Jordan L. Doman, David R. Liu   +3 more
openaire   +2 more sources

Adenine base editing in an adult mouse model of tyrosinaemia

, 2020
In contrast to traditional CRISPR-Cas9 homology-directed repair, base editing can correct point mutations without supplying a DNA-repair template. Here we show in a mouse model of tyrosinaemia that hydrodynamic tail-vein injection of plasmid DNA encoding
Yin, Hao, Zhu, Lihua Julie, Liu, David R., Anderson, Daniel G., Zafra, Maria Paz, Cao, Yueying, Dow, Lukas E., Xue, Wen, Jiang, Tingting, Song, Chun-Qing, Richter, Michelle, Rhym, Luke H., Koblan, Luke W., Doman, Jordan L., Schatoff, Emma M.   +14 more
core   +1 more source

Cytosine but not adenine base editor generates mutations in mice [PDF]

, 2019
ABSTRACT Deaminase base editing has emerged as a tool to install or correct point mutations in the genomes of living cells in a wide range of organisms and its ultimate success therapeutically depends on its accuracy. Here we have investigated the fidelity of cytosine base editor 4 (BE4) and adenine base editor (ABE) in mouse embryos ...
Lee, Hye Kyung, Smith, Harold E., Liu, Chengyu, Willi, Michaela, Hennighausen, Lothar   +4 more
openaire   +1 more source

Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction [PDF]

Nature Biotechnology, 2018
Base editors enable targeted single-nucleotide conversions in genomic DNA. Here we show that expression levels are a bottleneck in base-editing efficiency. We optimize cytidine (BE4) and adenine (ABE7.10) base editors by modification of nuclear localization signals (NLS) and codon usage, and ancestral reconstruction of the deaminase component.
Luke W Koblan, Jordan L Doman, Christopher Wilson, Jonathan M Levy, Tristan Tay, Gregory A Newby, Juan Pablo Maianti, Aditya Raguram, David R Liu   +8 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, Xingyun Huang, Xia Wang, Shixue Gou, Yanhui Liang, Fangbing Chen, Nan Li, Zhen Ouyang, Quanjun Zhang, Weikai Ge, Qin Jin, Hui Shi, Zhenpeng Zhuang, Xiaozhu Zhao, Meng Lian, Jiaowei Wang, Yinghua Ye, Longquan Quan, Han Wu, Kepin Wang, Liangxue Lai   +20 more
doaj   +1 more source

CABE-RY: A PAM-flexible dual-mutation base editor for reliable modeling of multi-nucleotide variants

Molecular Therapy: Nucleic Acids, 2021
Multi-nucleotide variants (MNVs) represent an important type of genetic variation and have biological and clinical significance. To simulate MNVs, we designed four dual-mutation base editors combining hA3A(Y130F), TadA8e(V106W), and protospacer adjacent ...
Wanyu Tao, Qing Liu, Shisheng Huang, Xin Wang, Shiyuan Qu, Junfan Guo, Danfeng Ou, Guanglei Li, Yu Zhang, Xiangmin Xu, Xingxu Huang   +10 more
doaj   +1 more source

Computational Approaches to Understand the Design of Adenine Base Editors [PDF]

, 2022
The most recent base editors, the adenine base editors (ABEs), catalyzes the conversionof A•T→G•C base pairs at precise genomic loci and were developed using extensive protein engineering and evolution starting from a RNA-editing enzyme, TadA.
Rallapalli, Kartik Lakshmi
core  

Voyage to minimal base editors

, 2022
Transposon-associated transposase B (TnpB) is the putative ancestor of Cas nucleases. A TnpB-based adenine base editor has now been developed that is small enough to be loaded into a single AAV vector without compromising editing activity ...
Sangsu Bae, Bae, Sangsu, Song, Beomjong, Beomjong Song   +3 more
core   +1 more source