Genome editing in human cells using CRISPR/Cas nucleases

Current Protocols in Molecular Biology

Volumn 2015, Issue , 2015, Pages

  Wyvekens, Nicolas ^a   Tsai, Shengdar Q ^a,b   Joung, J Keith ^a,b  

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

Author keywords

Cas9; CRISPR; FokI dCas9; Genome editing; Human cells; RNA guided FokI nucleases; Tru gRNA

Indexed keywords

CAS ENDONUCLEASE; CRISPR ASSOCIATED PROTEIN; ENDONUCLEASE; UNCLASSIFIED DRUG;

ARTICLE; CLONING; ENZYME SPECIFICITY; GENE EDITING; GENE EXPRESSION; GENE FREQUENCY; GENE MUTATION; GENOME ANALYSIS; HUMAN; HUMAN CELL; IN VITRO GENE TRANSFER; LIMIT OF QUANTITATION; PRIORITY JOURNAL; PROTEIN ENGINEERING; CELL LINE; CRISPR CAS SYSTEM; CYTOLOGY; GENE TARGETING; GENETIC RECOMBINATION; MOLECULAR BIOLOGY; PROCEDURES;

CELL LINE; CRISPR-CAS SYSTEMS; CYTOLOGICAL TECHNIQUES; GENE TARGETING; HUMANS; MOLECULAR BIOLOGY; RECOMBINATION, GENETIC;

EID: 85016241108     PISSN: 19343639     EISSN: 19343647     Source Type: Journal    
DOI: 10.1002/0471142727.mb3103s112     Document Type: Article

References (18)

1. Elbing, K. and Brent, R. 2002. Media preparation and bacteriological tools. Curr. Protoc. Mol. Biol. 59:1.1.1-1.1.7.
2. Fu, Y., Foden, J.A., Khayter, C., Maeder, M.L., Reyon, D., Joung, J.K., and Sander, J.D. 2013. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat. Biotechnol. 31:822-826. doi: 10.1038/nbt.2623.
3. Fu, Y., Sander, J.D., Reyon, D., Cascio, V.M., and Joung, J.K. 2014. Improving CRISPRCas nuclease specificity using truncated guide RNAs. Nat. Biotechnol. 32:279-284. doi: 10.1038/nbt.2808.
4. Guilinger, J.P., Thompson, D.B., and Liu, D.R. 2014. Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification. Nat. Biotechnol. 32:577-582. doi: 10.1038/nbt.2909.
5. Hsu, P.D., Scott, D.A., Weinstein, J.A., Ran, F.A., Konermann, S., Agarwala, V., Li, Y., Fine, E.J., Wu, X., Shalem, O., Cradick, T.J., Marraffini, L.A., Bao, G., and Zhang, F. 2013. DNA targeting specificity of RNA-guided Cas9 nucleases. Nat. Biotechnol. 31:827-832.
6. Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J.A., and Charpentier, E. 2012. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337:816-821.
7. Maddalo, D., Manchado, E., Concepcion, C.P., Bonetti, C., Vidigal, J.A., Han, Y.-C., Ogrodowski, P., Crippa, A., Rekhtman, N., de Stanchina, E., Lowe, S.W., and Ventura, A. 2014. In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system. Nature 516:423-427.
8. Mali, P., Aach, J., Stranges, P.B., Esvelt, K.M., Moosburner, M., Kosuri, S., Yang, L., and Church, G.M. 2013. Cas9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering. Nat. Biotechnol. 31:833-838.
9. Pattanayak, V., Lin, S., Guilinger, J.P., Ma, E., Doudna, J.A., and Liu, D.R. 2013. Highthroughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity. Nat. Biotechnol. 31:839-843.
10. Phelan, M.C. 2006. Techniques for mammalian cell tissue culture. Curr. Protoc. Mol. Biol. 74:A.3F.1-A.3F.18.
11. Reyon, D., Tsai, S.Q., Khayter, C., Foden, J.A., Sander, J.D., and Joung, J.K. 2012. FLASH assembly of TALENs for high-throughput genome editing. Nat. Biotechnol. 30:460-465.
12. Sander, J.D. and Joung, J.K. 2014. CRISPR-Cas systems for editing, regulating and targeting genomes. Nat. Biotechnol. 32:347-355.
13. Sander, J.D., Maeder, M.L., Reyon, D., Voytas, D.F., Joung, J.K., and Dobbs, D. 2010. ZiFiT (Zinc Finger Targeter): An updated zinc finger engineering tool. Nucleic Acids Res. 38:W462-W468.
14. Seidman, C.E., Struhl, K., Sheen, J., and Jessen, T. 1997. Introduction of plasmid DNA into cells. Curr. Protoc. Mol. Biol. 37:1.8.1-1.8.10.
15. Tsai, S.Q., Wyvekens, N., Khayter, C., Foden, J.A., Thapar, V., Reyon, D., Goodwin, M.J., Aryee, M.J., and Joung, J.K. 2014. Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing. Nat. Biotechnol. 32:569-576.
16. Tsai, S.Q., Zheng, Z., Nguyen, N.T., Liebers, M., Topkar, V.V., Thapar, V., Wyvekens, N., Khayter, C., Iafrate, A.J., Le, L.P., Aryee, M.J., and Joung, J.K. 2015. GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases. Nat. Biotechnol. 33:187-197.
17. Voytas, D. 2000. Agarose gel electrophoresis. Curr. Protoc. Mol. Biol. 51:2.5A.1-2.5A.9.
18. Wyvekens, N., Topkar, V.V., Khayter, C., Joung, J.K., and Tsai, S.Q. 2015. Dimeric CRISPR RNA-guided FokI-dCas9 nucleases directed by truncated gRNAs for highly specific genome editing. Hum. Gene Ther. 26:425-431.