Applications of genome editing tools in precision medicine research
Received date: 2016-11-23
Revised date: 2017-01-21
Online published: 2017-02-10
Supported by
the Shanghai Pujiang Talent Program(15PJ1409200);CAS Hundred Talent Program and the National Young Scientist Thousand Talent Program
The emergence of genome editing tools, such as the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 (Cas9) system, has enabled researchers to achieve somatic and germline genomic manipulations in cell lines and model organisms. Within a couple of years, genome editing is now being rapidly developed for multiple applications and widely used in biomedical researches, including creation of disease models with desired genetic mutations, screening in a high-throughput manner for drug resistance genes, and making appropriate editions to genes in vivo for disease treatment. All these applications have been facilitating the development of precision medicine research. In this review, we describe the use of genome editing technologies for a variety of research and translational applications in the precision medicine field. We also highlight some of the existing limitations or challenges as well as future directions.
Shuang Li,Yuanyuan Yang,Yan Qiu,Yanhao Chen,Luwei Xu,Qiurong Ding . Applications of genome editing tools in precision medicine research[J]. Hereditas(Beijing), 2017 , 39(3) : 177 -188 . DOI: 10.16288/j.yczz.16-395
| [1] | Collins FS, Varmus H. A new initiative on precision medicine. N Engl J Med, 2015, 372(9): 793-795. | |||
| [2] | Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD. Genome editing with engineered zinc finger nucleases. Nat Rev Genet, 2010, 11(9): 636-646. | |||
| [3] | Sun N, Zhao HM. Transcription activator-like effector nucleases (TALENs): a highly efficient and versatile tool for genome editing. Biotechnol Bioeng, 2013, 110(7): 1811-1821. | |||
| [4] | Hsu PD, Lander ES, Zhang F. Development and applications of CRISPR-Cas9 for genome engineering. Cell, 2014, 157(6): 1262-1278. | |||
| [5] | Zetsche B, Gootenberg JS, Abudayyeh OO, Slaymaker IM, Makarova KS, Essletzbichler P, Volz SE, Joung J, van der Oost J, Regev A, Koonin EV, Zhang F. Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system. Cell, 2015, 163(3): 759-771. | |||
| [6] | Fonfara I, Richter H, Bratovi? M, Le Rhun A, Charpentier E. The CRISPR-associated DNA-cleaving enzyme Cpf1 also processes precursor CRISPR RNA. Nature, 2016, 532(7600): 517-521. | |||
| [7] | Abudayyeh OO, Gootenberg JS, Konermann S, Joung J, Slaymaker IM, Cox DBT, Shmakov S, Makarova KS, Semenova E, Minakhin L, Severinov K, Regev A, Lander ES, Koonin EV, Zhang F. C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector.. Science, 2016, 353(6299): aaf5573. | |||
| [8] | East-Seletsky A, O'Connell MR, Knight SC, Burstein D, Cate JH, Tjian R, Doudna JA. Two distinct RNase activities of CRISPR-C2c2 enable guide-RNA processing and RNA detection. Nature, 2016, 538(7624): 270-273. | |||
| [9] | Xu S, Cao SS, Zou BJ, Yue YY, Gu C, Chen X, Wang P, Dong XH, Xiang Z, Li K, Zhu MS, Zhao QS, Zhou GH. An alternative novel tool for DNA editing without target sequence limitation: the structure-guided nuclease. Genome Biol, 2016, 17: 186. | |||
| [10] | Burstein D, Harrington LB, Strutt SC, Probst AJ, Anantharaman K, Thomas BC, Doudna JA, Banfield JF. New CRISPR-Cas systems from uncultivated microbes. Nature, 2016, 542(7640): 237-241. | |||
| [11] | Qi LS, Larson MH, Gilbert LA, Doudna JA, Weissman JS, Arkin AP, Lim WA. Repurposing CRISPR as an RNA- guided platform for sequence-specific control of gene expression. Cell, 2013, 152(5): 1173-1183. | |||
| [12] | Gilbert LA, Larson MH, Morsut L, Liu ZR, Brar GA, Torres SE, Stern-Ginossar N, Brandman O, Whitehead EH, Doudna JA, Lim WA, Weissman JS, Qi LS. CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes. Cell, 2013, 154(2): 442-451. | |||
| [13] | Cheng AW, Wang HY, Yang H, Shi LY, Katz Y, Theunissen TW, Rangarajan S, Shivalila CS, Dadon DB, Jaenisch R. Multiplexed activation of endogenous genes by CRISPR- on, an RNA-guided transcriptional activator system. Cell Res, 2013, 23(10)
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