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Molecular basis of engineered meganuclease targeting of the endogenous human RAG1 locus
2010
Nucleic Acids Research
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This is the first time that an engineered meganuclease variant targets the human RAG1 locus by stimulating homologous recombination in human cell lines up to 265 bp away from the cleavage site. ...
We have modified the homodimeric I-CreI endonuclease through protein engineering to target a specific DNA sequence within the human RAG1 gene. ...
ACKNOWLEDGEMENTS The authors would like to thank the beamline staff at the ESRF and SLS for helpful advice during data collection. ...
doi:10.1093/nar/gkq801
pmid:20846960
pmcid:PMC3025557
fatcat:yspb3htegvbifc4xbggebvtu7y
Meganucleases and Other Tools for Targeted Genome Engineering: Perspectives and Challenges for Gene Therapy
2011
Current Gene Therapy
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While a new generation of viral vectors is in the process of replacing the classical gamma-retrovirus-based approach, a number of strategies have emerged based on non-viral vectorization and/or targeted ...
Nuclease-based approaches, wherein an endonuclease is used to trigger site-specific genome editing, can significantly increase the percentage of targeted cells. ...
Re-engineering meganuclease specificity: example of a custom meganuclease targeting a sequence from the human XPC gene. (a) Comparison of the starting and final targets. ...
doi:10.2174/156652311794520111
pmid:21182466
pmcid:PMC3267165
fatcat:xpcdrq73cragthw7kbnpttmgam
Identification of Genes Regulating Gene Targeting by a High-Throughput Screening Approach
2011
Journal of Nucleic Acids
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We used this assay to explore a collection of siRNAs targeting 19,121 human genes. At the end of secondary screening, we had identified 64 genes for which knockdown affected nuclease-induced HGT. ...
Homologous gene targeting (HGT) is a precise but inefficient process for genome engineering. ...
For validation of the ability of the ATF7IP and EP300 siRNAs to increase HGT frequency at an endogenous locus in human cells, we used a meganuclease cleaving the human RAG1 locus described in a previous ...
doi:10.4061/2011/947212
pmid:21716659
pmcid:PMC3118287
fatcat:4nejp555kzernpapk5zb23bs3q
Meganucleases can restore the reading frame of a mutated dystrophin
2010
Gene Therapy
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These experiments are thus a proof of principle that MGNs that are adequately engineered to target appropriate sequences in the human dystrophin gene should be able to restore the normal reading frame ...
Meganucleases (MGNs) can be engineered to induce double-strand breaks (DSBs) at specific DNA sequences. ...
Conflict of interest Dr Pâques is the scientific director of Cellectis Inc. Thus, his salary is obtained from that company and he has shares in that company. ...
doi:10.1038/gt.2010.26
pmid:20393509
fatcat:o7enqz3dmjgb5iskpcv7nft4tm
Comparison of the Feasibility, Efficiency, and Safety of Genome Editing Technologies
2021
International Journal of Molecular Sciences
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Currently, no technology can be regarded as ideal or even applicable to every case as the context dictates the best approach for genetic modification within a target organism. ...
In this review, we implement a four-pillar framework (context, feasibility, efficiency, and safety) to assess the main genome editing platforms, as a basis for rational decision-making by an expanding ...
of RAG1 in 5.3% of
transfected cells
Plasmid in human 293H
cells
RAG1 MN (single-
chain I-CreI variant)
[25]
Gene insertion for repairing RAG1 in up to
6% of transfected cells
RAG1 MN (single ...
doi:10.3390/ijms221910355
pmid:34638696
fatcat:nvrlxxkbqbhbrhmhkdrrsxmjoi
Engineering altered protein–DNA recognition specificity
2018
Nucleic Acids Research
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The ease of re-engineering each system is related both to the modularity of the protein and the extent to which the proteins have evolved to be capable of readily modifying their recognition specificities ...
Nevertheless, progress has been made in the redesign of protein-DNA specificity, much of it driven by the development of engineered enzymes for genome modification. ...
the basis for the results of the engineering study described above. ...
doi:10.1093/nar/gky289
pmid:29718463
pmcid:PMC6007267
fatcat:zazdrfgoanagfby6yqvhydhzia
Gene Editing in Pluripotent Stem Cells and Their Derived Organoids
2021
Stem Cells International
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Are the tools for rewriting the code of life a boon to humanity or a Pandora's box? These technologies raise concerns regarding ethical issues, unexpected mutations, viral infection, etc. ...
The development of regenerative medicine technologies, regardless of whether it is PSCs or gene editing, is consistently met with controversy. ...
Acknowledgments This research was supported partly by the National Natural Science Foundation of China (82070638 and 81770621), JSPS KAKENHI (18H02866), and the Natural Science Foundation of Jiangsu Province ...
doi:10.1155/2021/8130828
pmid:34887928
pmcid:PMC8651378
fatcat:g7kj3nfrwrfltd4qxmyalw7iny
Advances in Genome Editing and Application to the Generation of Genetically Modified Rat Models
2021
Frontiers in Genetics
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The objective of this review is to provide a comprehensive description of the advantages and potential of rat models for addressing specific scientific questions and to characterize the best genome-engineering ...
Many genetically engineered rat models have emerged in the last two decades, and the advent of gene-specific nucleases has accelerated their generation in recent years. ...
AUTHOR CONTRIBUTIONS All authors performed the bibliographic research and participated in writing the manuscript. IA planned the review and secured the funding. ...
doi:10.3389/fgene.2021.615491
pmid:33959146
pmcid:PMC8093876
fatcat:6rygmc2dq5gorfeyyjiugg4wgq
Recent Advances and Improvements in the Biosafety of Gene Therapy
[chapter]
2011
Gene Therapy - Developments and Future Perspectives
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Thus far two engineered meganucleases cleaving unique genomic loci in the human XPC (Arnould et al., 2007) and Rag1 genes (Grizot et al., 2009 ) have been reported. ...
However, they have been used to bind vector sequences bearing their recognition elements and, fused with other endogenous DBPs, can be engineered to recognise elements in the human genome (Ivics et al ...
The aim of this book is to cover key aspects of existing problems in the field of development and future perspectives in gene therapy. ...
doi:10.5772/21733
fatcat:utqxetzf6vbphkgke3iyreqcd4
Cut and Paste - Using CRISPR/Cas9 to model RAG1 deficiency
2018
Zenodo
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The main aim of this thesis was to generate novel in vitro and in vivo models of RAG1 deficiency to study the mechanisms underlying the different RAG phenotypes. ...
However, phenotypic variability remains even between patients with mutations that affect the same region of RAG1, showing the same residual RAG1 activity using in vitro models. ...
Acknowledgments The authors thank K. Musunuru for his guidance in designing the gRNA to generate the Rag1-mutant mice and W. Yang and M. ...
doi:10.5281/zenodo.2625487
fatcat:4auhvifpincedcatl3ndxqdary
CRISPR to fix bad blood: a new tool in basic and clinical hematology
2019
Haematologica
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Advances in genome engineering in the last decade, particularly in the development of programmable nucleases, have made it possible to edit the genomes of most cell types precisely and efficiently. ...
We provide an overview of the applications of gene editing in experimental, preclinical and clinical hematology including interrogation of gene function, target identification and drug discovery and chimeric ...
Thus, researchers keen to perform gene editing require only a basic knowledge of molecular biology to design a targeting system against a locus of choice. ...
doi:10.3324/haematol.2018.211359
pmid:30923099
pmcid:PMC6518885
fatcat:unq36nyb7vb4lnkshxoeyoofxq
Concerted nicking of donor and chromosomal acceptor DNA promotes homology-directed gene targeting in human cells
2011
Nucleic Acids Research
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of the targeting vector. ...
Here, to investigate the role of nicked DNA molecules as HR-initiating substrates in human somatic cells, we devised a homology-directed gene targeting system based on exogenous donor and chromosomal target ...
ACKNOWLEDGEMENTS The authors thank Martijn Everts for PCR amplification and cloning of the p5 element. ...
doi:10.1093/nar/gkr1234
pmid:22189101
pmcid:PMC3333848
fatcat:emwm53oacfbhvlrwnkipmlemn4
Activation-Induced Cytidine Deaminase in Antibody Diversification and Chromosome Translocation
[chapter]
2012
Advances in Cancer Research
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DNA damage, rearrangement and mutation of the human genome are the basis of carcinogenesis and thought to be avoided at all costs. ...
Here we review the molecular basis of AID regulation, targeting, and initiation of CSR and SHM, as well as AID's role in generating chromosome translocations that contribute to lymphomagenesis. ...
The two groups that simultaneously developed this method both engineered I-SceI meganuclease sites at the IgH or the c-myc locus. ...
doi:10.1016/b978-0-12-394280-7.00005-1
pmid:22429855
pmcid:PMC4353630
fatcat:f4hyre5wc5a3jcnnqyqnan5thq
Homing endonucleases from mobile group I introns: discovery to genome engineering
2014
Mobile DNA
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This combination of molecular functions is mutually advantageous: the endonuclease activity allows surrounding introns and inteins to act as invasive DNA elements, while the splicing activity allows the ...
Several homing endonucleases have been completely redesigned and used for a variety of genome engineering applications. ...
Acknowledgments The work conducted in the authors laboratory, and the preparation of this review, were supported by a research grant from the NIH (R01 GM49857). ...
doi:10.1186/1759-8753-5-7
pmid:24589358
pmcid:PMC3943268
fatcat:guvzcedoozglpngb3bsqb4vehe
Humanization of Immunodeficient Animals for the Modeling of Transplantation, Graft Versus Host Disease and Regenerative Medicine
2020
Transplantation
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The humanization of animals is a powerful tool for the exploration of human disease pathogenesis in biomedical research, as well as for the development of therapeutic interventions with enhanced translational ...
These advances, together with ongoing developments in the incorporation of human transgenes and additional mutations in humanized mouse models, have expanded our opportunities to replicate aspects of human ...
[64] [65] [66] This is supported by a recent study in which expression of full-length human BAFF from cDNA in the endogenous mouse locus did not improve maturation of human B cells in HIS mice. 67 ...
doi:10.1097/tp.0000000000003177
pmid:32068660
pmcid:PMC7590965
fatcat:ddvzbordwrhjdi7sw2jzbmsxyy
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