Results 31 to 40 of about 515,050 (293)

A review of application of base editing for the treatment of inner ear disorders

Journal of Bio-X Research, 2020
. The development of applications for the CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated nuclease 9) system has increased greatly in recent years, especially in the area of gene therapy by efficient in vivo ...
Xingle Zhao, Zhuoer Sun, Wen Kang, Yong Tao, Hao Wu   +4 more
doaj   +1 more source

Base editing the mammalian genome [PDF]

Methods, 2019
Base editing is a powerful technology that enables programmable conversion of single nucleotides in the mammalian genome. Base editors consist of a partially active Cas9 nuclease (Cas9D10A) tethered to a natural or synthetic DNA modifying enzyme.
Emma M, Schatoff, Maria Paz, Zafra, Lukas E, Dow   +2 more
openaire   +2 more sources

Alternative splicing and extensive RNA editing of human TPH2 transcripts. [PDF]

, 2010
Brain serotonin (5-HT) neurotransmission plays a key role in the regulation of mood and has been implicated in a variety of neuropsychiatric conditions. Tryptophan hydroxylase (TPH) is the rate-limiting enzyme in the biosynthesis of 5-HT.
Bondy, Brigitta, Bondy, B., Baghai Thomas C., Brigitta Bondy, Peter Zill, Zill, P., Schüle Cornelius, Hammer, P., Grohmann Maik, Büttner, A., Walther Maria, Priller, Josef, Hammer Paul, Baghai, Thomas C., Maik Grohmann, Wolfgang Eisenmenger, Baghai, T., Priller Josef, Josef Priller, Walther, D.J., Eisenmenger, Wolfgang, Bader, Michael, Zill, Peter, Paul Hammer, Hammer, Paul, Rupprecht Rainer, Büttner, Andreas, Michael Bader, Andreas Büttner, Diego J Walther, Cornelius Schüle, Paulmann, Nils, Zill Peter, Bader Michael, Eisenmenger, W., Schüle, Cornelius, Maria Walther, Rainer Rupprecht, Thomas C Baghai, Paulmann Nils, Schuele, C., Walther, Diego J., Rupprecht, R., Nils Paulmann, Baghai, T.C., Eisenmenger Wolfgang, Walther, Maria, Grohmann, M., Bondy Brigitta, Walther Diego J., Bader, M., Priller, J., Paulmann, N., Büttner Andreas, Walther, M., Grohmann, Maik, Rupprecht, Rainer, Schüle, C., Walther, D., Buettner, A.   +59 more
core   +1 more source

Research progress in gene editing technology

Frontiers in Bioscience-Landmark, 2021
As a tool for modifying the genome, gene editing technology has developed rapidly in recent years, especially in the past two years. With the emergence of new gene editing technologies, such as transposon editing tools, numerous advancements have been
Sheng Huang, Yali Yan, Fei Su, Xiaorui Huang, Dandan Xia, Xinxin Jiang, Yinghui Dong, Ping Lv, Fangyuan Chen, Yanwei Lv   +9 more
doaj   +1 more source

sgBE: a structure-guided design of sgRNA architecture specifies base editing window and enables simultaneous conversion of cytosine and adenosine

Genome Biology, 2020
We present a base editing system, in which base editors are attached to different sites of sgRNA scaffold (sgBE). Each independent sgBE has its own specific editing pattern for a given target site.
Yanhong Wang, Lifang Zhou, Rui Tao, Nan Liu, Jie Long, Fengming Qin, Wenling Tang, Yang Yang, Qiang Chen, Shaohua Yao   +9 more
doaj   +1 more source

Multiplex precise base editing in cynomolgus monkeys

, 2021
© 2020, The Author(s). Common polygenic diseases result from compounded risk contributed by multiple genetic variants, meaning that simultaneous correction or introduction of single nucleotide variants is required for disease modeling and gene therapy ...
Wilde, Jonathan J, Tang, Yu, del Rosario, Ricardo CH, Zhang, Xiaohui, Lu, Hongyu, Jiang, Minqing, Huang, Yan, Li, Duanduan, Xu, Dongdong, Fang, Zhihao, Feng, Guoping, Zhang, Wenhui, Zheng, Zhanhong, Zhou, Yang, Aida, Tomomi, Huang, Qunshan, Ding, Chenhui, Baloch, Zulqurain, Yang, Shihua   +18 more
core   +1 more source

Expanding the Editing Window of Cytidine Base Editors With the Rad51 DNA-Binding Domain in Rice

Frontiers in Plant Science, 2022
Recently developed base editors provide a powerful tool for plant research and crop improvement. Although a number of different deaminases and Cas proteins have been used to improve base editors the editing efficiency, and editing window are still not ...
Chunjie Wei, Hao Liu, Wenwen Wang, Pengyu Luo, Qiuling Chen, Rou Li, Chong Wang, José Ramón Botella, Hui Zhang   +8 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

CRISPR base editing applications for identifying cancer-driving mutations [PDF]

, 2021
CRISPR base editing technology is a promising genome editing tool as (i) it does not require a DNA template to introduce mutations and (ii) it avoids creating DNA double-strand breaks, which can lead to unintended chromosomal alterations or elicit an ...
Herold, MJ, Pal, M
core   +1 more source

Base Editors for Citrus Gene Editing [PDF]

Frontiers in Genome Editing, 2021
Base editors, such as adenine base editors (ABE) and cytosine base editors (CBE), provide alternatives for precise genome editing without generating double-strand breaks (DSBs), thus avoiding the risk of genome instability and unpredictable outcomes caused by DNA repair.
Xiaoen Huang, Yuanchun Wang, Nian Wang
openaire   +4 more sources