CRISPR-Cas9 technology: applications and human disease modelling.
作者： Raul Torres-Ruiz , Sandra Rodriguez-Perales
DOI: 10.1093/BFGP/ELW025
关键词:
摘要: Genome engineering is a powerful tool for wide range of applications in biomedical research and medicine. The development the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 system has revolutionized field gene editing, thus facilitating efficient genome editing through creation targeted double-strand breaks almost any organism cell type. In addition, CRISPR-Cas9 technology been used successfully many other purposes, including regulation endogenous expression, epigenome live-cell labelling chromosomal loci, edition single-stranded RNA high-throughput screening. implementation increased number available technological alternatives studying function, enabling generation CRISPR-based disease models. Although mechanistic questions remain to be answered several challenges have yet addressed, use CRISPR-Cas9-based technologies will increase our knowledge processes their treatment near future.
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sci-hub.se 参考文章(185)
Xiquan Liang, Jason Potter, Shantanu Kumar, Yanfei Zou, Rene Quintanilla, Mahalakshmi Sridharan, Jason Carte, Wen Chen, Natasha Roark, Sridhar Ranganathan, Namritha Ravinder, Jonathan D. Chesnut, Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection Journal of Biotechnology. ,vol. 208, pp. 44- 53 ,(2015) , 10.1016/J.JBIOTEC.2015.04.024
Michael Boettcher, Michael T. McManus, Choosing the Right Tool for the Job: RNAi, TALEN, or CRISPR Molecular Cell. ,vol. 58, pp. 575- 585 ,(2015) , 10.1016/J.MOLCEL.2015.04.028
Youming Zhang, Frank Buchholz, Joep P.P. Muyrers, A. Francis Stewart, A new logic for DNA engineering using recombination in Escherichia coli Nature Genetics. ,vol. 20, pp. 123- 128 ,(1998) , 10.1038/2417
Benjamin P. Kleinstiver, Michelle S. Prew, Shengdar Q. Tsai, Ved V. Topkar, Nhu T. Nguyen, Zongli Zheng, Andrew P. W. Gonzales, Zhuyun Li, Randall T. Peterson, Jing-Ruey Joanna Yeh, Martin J. Aryee, J. Keith Joung, Engineered CRISPR-CAS9 nucleases with altered PAM specificity Nature. ,vol. 523, pp. 481- 485 ,(2016) , 10.1038/NATURE14592
Sayda M. Elbashir, Jens Harborth, Winfried Lendeckel, Abdullah Yalcin, Klaus Weber, Thomas Tuschl, Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells Nature. ,vol. 411, pp. 494- 498 ,(2001) , 10.1038/35078107
Dong-Jiunn Jeffery Truong, Karin Kühner, Ralf Kühn, Stanislas Werfel, Stefan Engelhardt, Wolfgang Wurst, Oskar Ortiz, Development of an intein-mediated split–Cas9 system for gene therapy Nucleic Acids Research. ,vol. 43, pp. 6450- 6458 ,(2015) , 10.1093/NAR/GKV601
Andrew Fire, SiQun Xu, Mary K. Montgomery, Steven A. Kostas, Samuel E. Driver, Craig C. Mello, Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans Nature. ,vol. 391, pp. 806- 811 ,(1998) , 10.1038/35888
Srinivasan Chandrasegaran, Dana Carroll, Origins of Programmable Nucleases for Genome Engineering Journal of Molecular Biology. ,vol. 428, pp. 963- 989 ,(2016) , 10.1016/J.JMB.2015.10.014
P Rouet, F Smih, M Jasin, Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease. Molecular and Cellular Biology. ,vol. 14, pp. 8096- 8106 ,(1994) , 10.1128/MCB.14.12.8096
Nicolas Wyvekens, Ved V. Topkar, Cyd Khayter, J. Keith Joung, Shengdar Q. Tsai, Dimeric CRISPR RNA-Guided FokI-dCas9 Nucleases Directed by Truncated gRNAs for Highly Specific Genome Editing Human Gene Therapy. ,vol. 26, pp. 425- 431 ,(2015) , 10.1089/HUM.2015.084