Results 41 to 50 of about 19,445 (257)

Adenine base editor correction of pathogenic variations associated with inherited retinal dystrophy in patient iPSC and retinal organoids. [PDF]

Mol Ther Nucleic Acids
Inherited retinal dystrophies (IRDs) are a group of incurable, genetically heterogeneous diseases that cause progressive degeneration of the retina, leading to the loss of vision.
Leung A, Perdigão PRL, Sacristan-Reviriego A, Sladen PE, Aguzzi EA, Rezek FO, Ziaka K, Guarascio R, Hau KL, Cheetham ME, van der Spuy J.   +10 more
europepmc   +2 more sources

A novel base editor SpRY-ABE8eF148A mediates efficient A-to-G base editing with a reduced off-target effect

Molecular Therapy: Nucleic Acids, 2023
Adenine base editors (ABEs) can mediate two transition mutations, A-to-G and T-to-C, which are suitable for repairing G·C-to-T·A pathogenic variants, the most significant human pathogenic variant.
Guo Li, Yaxian Cheng, Yeqiu Li, Hongru Ma, Zhongji Pu, Sa Li, Yiqiang Zhao, Xingxu Huang, Yuan Yao   +8 more
doaj   +1 more source

Developing high-efficiency base editors by combining optimized synergistic core components with new types of nuclear localization signal peptide

Crop Journal, 2020
The clustered regularly interspaced short palindromic repeats (CRISPR)–CRISPR-associated protein (Cas) system has been widely used for genome editing. In this system, the cytosine base editor (CBE) and adenine base editor (ABE) allow generating precise ...
Feipeng Wang, Chengwei Zhang, Wen Xu, Shuang Yuan, Jinling Song, Lu Li, Jiuran Zhao, Jinxiao Yang   +7 more
doaj   +1 more source

Efficient precise in vivo base editing in adult dystrophic mice

Nature Communications, 2021
Base editing is one approach used to correct mutations causing cause Duchenne muscular dystrophy (DMD), but limitations are in the requirement for a specific PAM motif and the large size beyond the packaging capacity of adeno-associated virus (AAV). Here,
Li Xu, Chen Zhang, Haiwen Li, Peipei Wang, Yandi Gao, Nahush A. Mokadam, Jianjie Ma, W. David Arnold, Renzhi Han   +8 more
doaj   +1 more source

CRISPR base editing and prime editing: DSB and template-free editing systems for bacteria and plants

Synthetic and Systems Biotechnology, 2020
CRISPR-Cas (Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR associated) has been extensively exploited as a genetic tool for genome editing.
Abdullah, Zhengzheng Jiang, Xulin Hong, Shun Zhang, Ruilian Yao, Yi Xiao   +5 more
doaj   +1 more source

Development of a Simple and Quick Method to Assess Base Editing in Human Cells

Molecular Therapy: Nucleic Acids, 2020
Base editing is a form of genome editing that can directly convert a single base (C or A) to another base (T or G), which is of great potential in biomedical applications.
Xiujuan Lv, Kairui Qiu, Tianxiang Tu, Xiaoxue He, Yuchen Peng, Jinbin Ye, Junhao Fu, Ruzhi Deng, Yuqin Wang, Jinyu Wu, Changbao Liu, Junzhao Zhao, Feng Gu   +12 more
doaj   +1 more source

Protocol for targeted modification of the rice genome using base editing

STAR Protocols, 2022
Summary: Base editing is a precision genome-editing approach that is widely utilized to generate single-nucleotide variants (SNVs) in genomes. Here, we present a protocol to perform targeted adenine (A)-to-guanine (G) substitution in rice using adenine ...
Meixia Wang, Fang Yan, Huanbin Zhou
doaj   +1 more source

Examination of the Cell Cycle Dependence of Cytosine and Adenine Base Editors

Frontiers in Genome Editing, 2022
Base editors (BEs) are genome editing agents that install point mutations with high efficiency and specificity. Due to their reliance on uracil and inosine DNA damage intermediates (rather than double-strand DNA breaks, or DSBs), it has been hypothesized that BEs rely on more ubiquitous DNA repair pathways than DSB-reliant genome editing methods, which
Cameron A. Burnett, Ashley T. Wong, Carlos A. Vasquez, Colleen A. McHugh, Gene W. Yeo, Gene W. Yeo, Alexis C. Komor   +6 more
openaire   +5 more sources

Targeting fidelity of adenine and cytosine base editors in mouse embryos [PDF]

Nature Communications, 2018
AbstractBase editing directly converts a target base pair into a different base pair in the genome of living cells without introducing double-stranded DNA breaks. While cytosine base editors (CBE) and adenine base editors (ABE) are used to install and correct point mutations in a wide range of organisms, the extent and distribution of off-target edits ...
Hye Kyung Lee, Michaela Willi, Shannon M. Miller, Sojung Kim, Chengyu Liu, David R. Liu, Lothar Hennighausen   +6 more
openaire   +3 more sources

One-step generation of tumor models by base editor multiplexing in adult stem cell-derived organoids [PDF]

, 2023
Optimization of CRISPR/Cas9-mediated genome engineering has resulted in base editors that hold promise for mutation repair and disease modeling. Here, we demonstrate the application of base editors for the generation of complex tumor models in human ASC ...
Gijs van Son, Amanda Andersson-Rolf, Johan H. van Es, Lucca L. M. Derks, Hans Clevers, Sarina Harshuk-Shabso, Delilah Hendriks, Ruben van Boxtel, Flavia Peci, Paul Schürmann, Harry Begthel, Susana M. Chuva de Sousa Lopes, Ninouk Akkerman, Maarten H. Geurts, Matteo G. Boretto, Martina Celotti, Shashank Gandhi   +16 more
core   +2 more sources