Cornerstones of CRISPR-Cas in drug discovery and therapy

被引：346

作者：

Fellmann, Christof ^{[1
]}

Cowen, Benjamin C. ^{[1
,2
]}

Lin, Pei-Chun ^{[1
,2
,3
]}

Doudna, Jennifer A. ^{[1
,4
,5
,6
,7
]}

Corn, Jacob E. ^{[1
,2
]}

机构：

[1] Univ Calif Berkeley, Dept Mol & Cell Biol, 229 Stanley Hall, Berkeley, CA 94720 USA

[2] Univ Calif Berkeley, Innovat Genom Inst, Berkeley, CA 94720 USA

[3] Univ Calif San Francisco, Helen Diller Family Comprehens Canc Ctr, San Francisco, CA 94158 USA

[4] Univ Calif Berkeley, Dept Chem, Berkeley, CA 94720 USA

[5] Univ Calif Berkeley, Howard Hughes Med Inst, Berkeley, CA 94720 USA

[6] Univ Calif Berkeley, Li Ka Shing Biomed & Hlth Sci Ctr, Berkeley, CA 94720 USA

[7] Lawrence Berkeley Natl Lab, MBIB Div, Berkeley, CA 94720 USA

来源：

NATURE REVIEWS DRUG DISCOVERY | 2017年 / 16卷 / 02期

基金：

美国国家卫生研究院;

关键词：

EMBRYONIC STEM-CELLS; HEMATOPOIETIC STEM/PROGENITOR CELLS; GENOME-EDITING SPECIFICITY; ZINC-FINGER NUCLEASES; VIRUS ENTRY REQUIRES; DOUBLE-STRAND BREAKS; HUMAN CANCER-CELLS; OFF-TARGET SITES; IN-VIVO; T-CELLS;

D O I：

10.1038/nrd.2016.238

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

The recent development of CRISPR-Cas systems as easily accessible and programmable tools for genome editing and regulation is spurring a revolution in biology. Paired with the rapid expansion of reference and personalized genomic sequence information, technologies based on CRISPR-Cas are enabling nearly unlimited genetic manipulation, even in previously difficult contexts, including human cells. Although much attention has focused on the potential of CRISPR-Cas to cure Mendelian diseases, the technology also holds promise to transform the development of therapies to treat complex heritable and somatic disorders. In this Review, we discuss how CRISPR-Cas can affect the next generation of drugs by accelerating the identification and validation of high-value targets, uncovering high-confidence biomarkers and developing differentiated breakthrough therapies. We focus on the promises, pitfalls and hurdles of this revolutionary gene-editing technology, discuss key aspects of different CRISPR-Cas screening platforms and offer our perspectives on the best practices in genome engineering.

引用

页码：89 / 100

页数：12

共 159 条

[1] Genomic Copy Number Dictates a Gene-Independent Cell Response to CRISPR/Cas9 Targeting [J].

Aguirre, Andrew J. ;

Meyers, Robin M. ;

Weir, Barbara A. ;

Vazquez, Francisca ;

Zhang, Cheng-Zhong ;

Ben-David, Uri ;

Cook, April ;

Ha, Gavin ;

Harrington, William F. ;

Doshi, Mihir B. ;

Kost-Alimova, Maria ;

Gill, Stanley ;

Xu, Han ;

Ali, Levi D. ;

Jiang, Guozhi ;

Pantel, Sasha ;

Lee, Yenarae ;

Goodale, Amy ;

Cherniack, Andrew D. ;

Oh, Coyin ;

Kryukov, Gregory ;

Cowley, Glenn S. ;

Garraway, Levi A. ;

Stegmaier, Kimberly ;

Roberts, Charles W. ;

Golub, Todd R. ;

Meyerson, Matthew ;

Root, David E. ;

Tsherniak, Aviad ;

Hahn, William C. .

CANCER DISCOVERY, 2016, 6 (08) :914-929

[2]

[Anonymous], 2015, BLOOD

[3] Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases [J].

Bae, Sangsu ;

Park, Jeongbin ;

Kim, Jin-Soo .

BIOINFORMATICS, 2014, 30 (10) :1473-1475

[4] The Cancer Cell Line Encyclopedia enables predictive modelling of anticancer drug sensitivity [J].

Barretina, Jordi ;

Caponigro, Giordano ;

Stransky, Nicolas ;

Venkatesan, Kavitha ;

Margolin, Adam A. ;

Kim, Sungjoon ;

Wilson, Christopher J. ;

Lehar, Joseph ;

Kryukov, Gregory V. ;

Sonkin, Dmitriy ;

Reddy, Anupama ;

Liu, Manway ;

Murray, Lauren ;

Berger, Michael F. ;

Monahan, John E. ;

Morais, Paula ;

Meltzer, Jodi ;

Korejwa, Adam ;

Jane-Valbuena, Judit ;

Mapa, Felipa A. ;

Thibault, Joseph ;

Bric-Furlong, Eva ;

Raman, Pichai ;

Shipway, Aaron ;

Engels, Ingo H. ;

Cheng, Jill ;

Yu, Guoying K. ;

Yu, Jianjun ;

Aspesi, Peter, Jr. ;

de Silva, Melanie ;

Jagtap, Kalpana ;

Jones, Michael D. ;

Wang, Li ;

Hatton, Charles ;

Palescandolo, Emanuele ;

Gupta, Supriya ;

Mahan, Scott ;

Sougnez, Carrie ;

Onofrio, Robert C. ;

Liefeld, Ted ;

MacConaill, Laura ;

Winckler, Wendy ;

Reich, Michael ;

Li, Nanxin ;

Mesirov, Jill P. ;

Gabriel, Stacey B. ;

Getz, Gad ;

Ardlie, Kristin ;

Chan, Vivien ;

Myer, Vic E. .

NATURE, 2012, 483 (7391) :603-607

[5] An Erythroid Enhancer of BCL11A Subject to Genetic Variation Determines Fetal Hemoglobin Level [J].

Bauer, Daniel E. ;

Kamran, Sophia C. ;

Lessard, Samuel ;

Xu, Jian ;

Fujiwara, Yuko ;

Lin, Carrie ;

Shao, Zhen ;

Canver, Matthew C. ;

Smith, Elenoe C. ;

Pinello, Luca ;

Sabo, Peter J. ;

Vierstra, Jeff ;

Voit, Richard A. ;

Yuan, Guo-Cheng ;

Porteus, Matthew H. ;

Stamatoyannopoulos, John A. ;

Lettre, Guillaume ;

Orkin, Stuart H. .

SCIENCE, 2013, 342 (6155) :253-257

[6] Efficient method to generate single-copy transgenic mice by site-specific integration in embryonic stem cells [J].

Beard, C ;

Hochedlinger, K ;

Plath, K ;

Wutz, A ;

Jaenisch, R .

GENESIS, 2006, 44 (01) :23-28

[7] Integrated genomic analyses of ovarian carcinoma [J].

Bell, D. ;

Berchuck, A. ;

Birrer, M. ;

Chien, J. ;

Cramer, D. W. ;

Dao, F. ;

Dhir, R. ;

DiSaia, P. ;

Gabra, H. ;

Glenn, P. ;

Godwin, A. K. ;

Gross, J. ;

Hartmann, L. ;

Huang, M. ;

Huntsman, D. G. ;

Iacocca, M. ;

Imielinski, M. ;

Kalloger, S. ;

Karlan, B. Y. ;

Levine, D. A. ;

Mills, G. B. ;

Morrison, C. ;

Mutch, D. ;

Olvera, N. ;

Orsulic, S. ;

Park, K. ;

Petrelli, N. ;

Rabeno, B. ;

Rader, J. S. ;

Sikic, B. I. ;

Smith-McCune, K. ;

Sood, A. K. ;

Bowtell, D. ;

Penny, R. ;

Testa, J. R. ;

Chang, K. ;

Dinh, H. H. ;

Drummond, J. A. ;

Fowler, G. ;

Gunaratne, P. ;

Hawes, A. C. ;

Kovar, C. L. ;

Lewis, L. R. ;

Morgan, M. B. ;

Newsham, I. F. ;

Santibanez, J. ;

Reid, J. G. ;

Trevino, L. R. ;

Wu, Y. -Q. ;

Wang, M. .

NATURE, 2011, 474 (7353) :609-615

[8] Gene essentiality and synthetic lethality in haploid human cells [J].

Blomen, Vincent A. ;

Majek, Peter ;

Jae, Lucas T. ;

Bigenzahn, Johannes W. ;

Nieuwenhuis, Joppe ;

Staring, Jacqueline ;

Sacco, Roberto ;

van Diemen, Ferdy R. ;

Olk, Nadine ;

Stukalov, Alexey ;

Marceau, Caleb ;

Janssen, Hans ;

Carette, Jan E. ;

Bennett, Keiryn L. ;

Colinge, Jacques ;

Superti-Furga, Giulio ;

Brummelkamp, Thijn R. .

SCIENCE, 2015, 350 (6264) :1092-1096

[9]

Bolukbasi MF, 2016, NAT METHODS, V13, P41, DOI [10.1038/NMETH.3684, 10.1038/nmeth.3684]

[10]

Cancer Genome Atlas Research Networks (231 collaborators), 2013, NATURE, V455, P1061

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