Heritable gene-targeting with gRNA/Cas9 in rats

Hu, Xinli; Chang, Nannan; Wang, Xuelian; Zhou, Fengyun; Zhou, Xiaohai; Zhu, Xiaojun; Xiong, Jing-Wei

doi:10.1038/cr.2013.141

Cited by 49 publications

(40 citation statements)

References 15 publications

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“…These studies suggested that one to six mismatches are tolerated depending on the position and that bases 8-14 at the 3′ end of a target sequence is the critical region for recognition by gRNA. However, in contrast to cell-based experiments, most reports show that genetically-modifi ed animals produced by RNA injection have low off-target frequency Yang et al 2013 ;Hu et al 2013 ;Li et al 2013a , b ;Bassett et al 2013 ;Friedland et al 2013 ;Hwang et al 2013 ). Furthermore, we have not detected any off-target site mutations in over thirty KO founder rats (Yoshimi et al 2014 ).…”

Section: Specifi City Of Crispr To Targeted Sitescontrasting

confidence: 61%

Engineered Nucleases Lead to Genome Editing Revolution in Rats

Yoshimi

Kaneko

Voigt

et al. 2014

Targeted Genome Editing Using Site-Specific Nucleases

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Sequence-specifi c endonucleases, such as zinc fi nger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas), allow simple generation of genetically modifi ed animals. We utilized ZFNs and TALENs to generate several knockout rat models of human diseases. Furthermore, CRISPR/ Cas9 used in conjunction with single-stranded oligodeoxyribonucleotides enabled us to generate several types of targeted knockin animal, such as single nucleotide polymorphism substitution, short DNA fragment integration, and large DNA fragment elimination. These powerful technologies have opened new doors for genome manipulation that enable the engineering of animal models of human disease and potential therapeutic applications.

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Section: Specifi City Of Crispr To Targeted Sitescontrasting

confidence: 61%

Engineered Nucleases Lead to Genome Editing Revolution in Rats

Yoshimi

Kaneko

Voigt

et al. 2014

Targeted Genome Editing Using Site-Specific Nucleases

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“…Recently, the CRISPR technology was selected as one of the ten breakthroughs of the year by the journal Science ( Genetic microsurgery for the masses 2013 ). Within just 1 year, applications of CRISPRmediated genome modifi cation were described in more than 50 publications using a wide range of hosts, such as human cells (Jinek et al 2012(Jinek et al , 2013Cong et al 2013 ;Mali et al 2013a ;Cho et al 2013 ;Hou et al 2013 ;, rat (Hu et al 2013 ;Li et al 2013a ), zebrafi sh (Auer et al 2014 ;Chang et al 2013 ;Hwang et al 2013 ;Jao et al 2013 ;Blackburn et al 2013 ), fruit fl y (Bassett et al 2013 ;Gratz et al 2013 ;Baena-Lopez et al 2013 ), nematode (Friedland et al 2013 ), mouse (Cong et al 2013 ;Li et al 2013a ;Yang et al 2013 ), yeast (Dicarlo et al 2013 ), bacteria (Jiang et al 2013a ), plants (Mao et al 2013 ;Miao et al 2013 ;Nekrasov et al 2013 ;Shan et al 2013 ;Upadhyay et al 2013 ;Xie and Yang 2013 ;Jiang et al 2013b ;Li et al 2013b ;Liang et al 2014 ), and many other organisms. Mutation rates obtained with the CRISPR system are comparable to those observed with ZFNs and TALENs, or in many cases even higher in stably transformed plants (Feng et al 2013 ).…”

Section: Introductionmentioning

confidence: 99%

Developing CRISPR Technology in Major Crop Plants

Chen

Gao

2015

Advances in New Technology for Targeted Modification of Plant Genomes

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Targeted genome modifi cation (TGM) by sequence-specifi c nucleases (SSNs) is a powerful tool for elucidating gene function and improving crops. Very recently, clustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated (Cas) system-based RNA-guided endonucleases have been added to the SSN toolbox. TGMs generated by this system rely on a synthetic single guide RNA (sgRNA) to direct the Cas9 protein to cleave a predetermined DNA sequence. Unlike previous SSNs, CRISPR provides a simple, cost-effective and versatile approach to multiplex genome engineering. In this review, we describe the molecular mechanisms involved in the CRISPR system, and summarize and discuss the applications of this technology in plant genome engineering.

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“…For instance, its efficiency is higher than HR, which makes it feasible to modify human cells; and it is also considerably easier to design guide RNA constructs than ZFNs and TALENs, making it suitable to perform genome-scale genes knockout screening. The gene knockout capabilities of the CRISPR-Cas9 system have been successfully shown in many model organisms, such as mice Wu et al 2013;Yang et al 2013b), rats (Hu et al 2013;Li et al 2013;Ma et al 2014), pigs (Hai et al 2014), and even monkeys . The CRISPR-Cas9 system can be used to correct a genetic disease in mouse via HDR based on an exogenously supplied oligonucleotide or the endogenous Wilms' tumor (WT) allele by coinjection into zygotes of Cas9 mRNA and an sgRNA targeting the mutant allele .…”

Section: Genome-wide Screening Via Crispr-cas9 For Diploid Pluripotenmentioning

confidence: 99%

Next-Generation Models of Human Cardiogenesis via Genome Editing

Lian

et al. 2014

Cold Spring Harbor Perspectives in Medicine

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Cardiogenesis is one of the earliest and most important steps during human development and is orchestrated by discrete families of heart progenitors, which build distinct regions of the fetal heart. For the past decade, a lineage map for the distinct subsets of progenitors that generate the embryonic mammalian heart has begun to lay a foundation for the development of new strategies for rebuilding the adult heart after injury, an unmet clinical need for the vast majority of patients with end-stage heart failure who are not heart transplant recipients. The studies also have implications for the root causes of congenital heart disease, which affects 1 in 50 live births, the most prevalent malformations in children. Although much of this insight has been generated in murine models, it is becoming increasingly clear that there can be important divergence with principles and pathways for human cardiogenesis, as well as for regenerative pathways. The development of human stem cell models, coupled with recent advances in genome editing with RNA-guided endonucleases, offers a new approach for the primary study of human cardiogenesis. In addition, application of the technology to the in vivo setting in large animal models, including nonhuman primates, has opened the door to genome-edited large animal models of adult and congenital heart disease, as well as a detailed mechanistic dissection of the more diverse and complex set of progenitor families and pathways, which guide human cardiogenesis. Implications of this new technology for a new generation of human-based, genetically tractable systems are discussed, along with potential therapeutic applications. A DECADE OF ADVANCES IN MURINE CARDIOGENESISO ver the past decade, major advances in our understanding of conserved principles and pathways for cardiogenesis have been made through genetically engineered mouse models of cardiogenesis. The assumption has been that the major underpinnings of such a conserved and essential organ system would have parallels in human organogenesis. The approach has been largely based on gene knockout apEditors:

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Heritable gene-targeting with gRNA/Cas9 in rats

Cited by 49 publications

References 15 publications

Engineered Nucleases Lead to Genome Editing Revolution in Rats

Engineered Nucleases Lead to Genome Editing Revolution in Rats

Developing CRISPR Technology in Major Crop Plants

Next-Generation Models of Human Cardiogenesis via Genome Editing

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