Results 61 to 70 of about 15,403 (255)

Two Compact Cas9 Ortholog-Based Cytosine Base Editors Expand the DNA Targeting Scope and Applications In Vitro and In Vivo

Frontiers in Cell and Developmental Biology, 2022
CRISPR/Cas9-based base editing tools enable precise genomic installation and hold great promise for gene therapy, whereas the big size of Cas9 nucleases and its reliability on specific protospacer adjacent motif (PAM) sequences as well as target site ...
Susu Wu, Liping Li, Min Li, Shiyu Sun, Shiyu Sun, Yuting Zhao, Xiaowen Xue, Feiyu Chen, Jingli Zhong, Junfan Guo, Junfan Guo, Qianhui Qu, Xiongjun Wang, Zhen Liu, Zhen Liu, Yunbo Qiao   +15 more
doaj   +1 more source

Genome Engineering in Plant Using an Efficient CRISPR-xCas9 Toolset With an Expanded PAM Compatibility

Frontiers in Genome Editing, 2020
The CRISPR-Cas9 system enables simple, rapid, and effective genome editing in many species. Nevertheless, the requirement of an NGG protospacer adjacent motif (PAM) for the widely used canonical Streptococcus pyogenes Cas9 (SpCas9) limits the potential ...
Chengwei Zhang, Guiting Kang, Xinxiang Liu, Si Zhao, Shuang Yuan, Lu Li, Yongxing Yang, Feipeng Wang, Xiang Zhang, Jinxiao Yang   +9 more
doaj   +1 more source

Precise genome editing with base editors

Medical Review, 2023
Single-nucleotide variants account for about half of known pathogenic genetic variants in human. Genome editing strategies by reversing pathogenic point mutations with minimum side effects have great therapeutic potential and are now being actively ...
Liu Hongcai, Zhu Yao, Li Minjie, Gu Zhimin   +3 more
doaj   +1 more source

Engineering of cytosine base editors with DNA damage minimization and editing scope diversification

Nucleic Acids Research, 2023
Abstract Cytosine base editors (CBEs), which enable precise C-to-T substitutions, have been restricted by potential safety risks, including DNA off-target edits, RNA off-target edits and additional genotoxicity such as DNA damages induced by double-strand breaks (DSBs).
Bo Yuan, Shuqian Zhang, Liting Song, Jinlong Chen, Jixin Cao, Jiayi Qiu, Zilong Qiu, Jingqi Chen, Xing-Ming Zhao, Tian-Lin Cheng   +9 more
openaire   +2 more sources

Base Editor Scanning Reveals Activating Mutations of DNMT3A

DNA methyltransferase 3A (DNMT3A) is a de novo cytosine methyltransferase responsible for establishing proper DNA methylation during mammalian development.
Whitney K. Lieberman (16859980), Jessica K. Liang (16859977), Nicholas Z. Lue (12086211), Derek D. Hwang (16859983), Emma M. Garcia (16859974), Brian B. Liau (1746394), James C. Woods (16859986)   +6 more
core   +4 more sources

Genome- and transcriptome-wide off-target analyses of an improved cytosine base editor

Plant Physiology, 2021
Abstract Cytosine base editors (CBEs) are the promising tools for precise genome editing in plants. It is important to investigate potential off-target effects of an efficient CBE at the genome and transcriptome levels in a major crop. Based on comparison of five cytidine deaminases and two different promoters for expressing single-guide
Linnell Bentley Randall, Simon Sretenovic, Yuechao Wu, Desuo Yin, Tao Zhang, Joyce Van Eck, Yiping Qi   +6 more
openaire   +2 more sources

Molecular Mechanism of the Cytosine CRISPR Base Editing Process and the Roles of Translesion DNA Polymerases

, 2021
CRISPR-mediated base editing causes damage to DNA, mainly uracil, apurinic/apyrimidinic (AP) sites, and nicks, which require various DNA repair mechanisms to complete the base conversion process.
Hongwu Ma, Shiming Pu (6991505), Chunzhi Zhang, Zuping Zhou, Xueli Zhang, Guo Jiang, Jie Yang, Xiaoping Liao, Xuxu Chen (8409636), Xuxu Chen, Dongdong Zhao (1681897), Yanhe Ma, Dongdong Zhao, Shiming Pu, Jie Wang, Jie Wang (16762), Changhao Bi (1764364), Xiaoping Liao (592112), Guo Jiang (4583215), Ju Li (354128), Yanhe Ma (28802), Zuping Zhou (354844), Xueli Zhang (383859), Hongwu Ma (439532), Changhao Bi, Jie Yang (121778), Chunzhi Zhang (3455954), Siwei Li, Ju Li, Siwei Li (144874), Yaqiu Li (4862851), Yaqiu Li   +31 more
core   +1 more source

Adeno‐associated virus serotype 2 capsid variants for improved liver‐directed gene therapy

Hepatology, EarlyView., 2022
Abstract Background and Aims Current liver‐directed gene therapies look for adeno‐associated virus (AAV) vectors with improved efficacy. With this background, capsid engineering is explored. Whereas shuffled capsid library screenings have resulted in potent liver targeting variants with one first vector in human clinical trials, modifying natural ...
Nadja Meumann, Marti Cabanes‐Creus, Moritz Ertelt, Renina Gale Navarro, Julie Lucifora, Qinggong Yuan, Karin Nien‐Huber, Ahmed Abdelrahman, Xuan‐Khang Vu, Liang Zhang, Ann‐Christin Franke, Christian Schmithals, Albrecht Piiper, Annabelle Vogt, Maria Gonzalez‐Carmona, Jochen T. Frueh, Evelyn Ullrich, Philip Meuleman, Steven R. Talbot, Margarete Odenthal, Michael Ott, Erhard Seifried, Clara T. Schoeder, Joachim Schwäble, Leszek Lisowski, Hildegard Büning   +25 more
wiley   +1 more source

CRISPR/Cas9-Mediated Cytosine Base Editing Using an Improved Transformation Procedure in Melon (Cucumis melo L.)

, 2023
International audienceMelon is a recalcitrant plant for stable genetic transformation. Various protocols have been tried to improve melon transformation efficiency; however, it remains significantly low compared to other plants such as tomato.
Dogimont, Catherine, Shojaeiyan, Abdolali, Hadi Shirazi Parsa, Sabet, Mohammad Sadegh, Abdelhafid Bendahmane, Ahmad Moieni, Moieni, Ahmad, Abdolali Shojaeiyan, Bendahmane, Abdelhafid, Mohammad Sadegh Sabet, Adnane Boualem, Boualem, Adnane, Shirazi Parsa, Hadi, Catherine Dogimont   +13 more
core   +1 more source

Engineered Sdd7 cytosine base editors with enhanced specificity

Nature Communications
Cytosine base editors (CBEs) revolutionize genome editing by enabling precise C-to-T conversions without double-strand breaks. Sdd7, a recently developed cytosine deaminase, exhibits high activity across a broad protospacer range but induces unintended off-target effects, including bystander mutations within and upstream of the protospacer and both ...
Hye-Yeon Hwang, Minyoung Lee, Hwalin Yi, Cheong Seok, Kayeong Lim, Yi Rang Na, Jong-Sun Kang, Jae-Hyun Park, Daesik Kim   +8 more
openaire   +2 more sources