Advances in therapeutic CRISPR/Cas9 genome editing

Translational Research

Volumn 168, Issue , 2016, Pages 15-21

  Savić, Nataša ^a   Schwank, Gerald ^a  

^a ETH ZURICH   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

CRISPR ASSOCIATED PROTEIN; CRISPR ASSOCIATED PROTEIN 9; UNCLASSIFIED DRUG; CASPASE 9;

CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; CRISPR CAS SYSTEM; EX VIVO STUDY; GENE THERAPY; GENOME; HUMAN; IN VIVO STUDY; NONHUMAN; PRIORITY JOURNAL; REVIEW; ZYGOTE; ANIMAL; GENE EXPRESSION REGULATION; GENETICS; PHYSIOLOGY;

ANIMALS; CASPASE 9; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS; CRISPR-CAS SYSTEMS; GENE EXPRESSION REGULATION; GENETIC THERAPY;

EID: 84973473524     PISSN: 19315244     EISSN: 18781810     Source Type: Journal    
DOI: 10.1016/j.trsl.2015.09.008     Document Type: Review

References (51)

1. 1 Wiedenheft, B., Sternberg, S.H., Doudna, J.A., RNA-guided genetic silencing systems in bacteria and archaea. Nature 482 (2012), 331–338.
2. 2 Doudna, J.A., Charpentier, E., The new frontier of genome engineering with CRISPR-Cas9. Science, 346, 2014, 1258096.
3. 3 Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J.A., Charpentier, E., A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337 (2012), 816–821.
4. 4 Mali, P., Yang, L., Esvelt, K.M., et al. RNA-guided human genome engineering via Cas9. Science 339 (2013), 823–826.
5. 5 Barnes, D.E., Non-homologous end joining as a mechanism of DNA repair. Curr Biol 11 (2001), R455–R457.
6. 6 Van den Bosch, M., Lohman, P.H., Pastink, A., DNA double-strand break repair by homologous recombination. Biol Chem 383 (2002), 873–892.
7. 7 Joung, J.K., Sander, J.D., TALENs: a widely applicable technology for targeted genome editing. Nat Rev Mol Cell Biol 14 (2013), 49–55.
8. 8 Urnov, F.D., Rebar, E.J., Holmes, M.C., Zhang, H.S., Gregory, P.D., Genome editing with engineered zinc finger nucleases. Nat Rev Genet 11 (2010), 636–646.
9. 9 Cho, S.W., Kim, S., Kim, J.M., Kim, J.S., Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat Biotechnol 31 (2013), 230–232.
10. 10 Cong, L., Ran, F.A., Cox, D., et al. Multiplex genome engineering using CRISPR/Cas systems. Science 339 (2013), 819–823.
11. 11 Ding, Q., Regan, S.N., Xia, Y., Oostrom, L.A., Cowan, C.A., Musunuru, K., Enhanced efficiency of human pluripotent stem cell genome editing through replacing TALENs with CRISPRs. Cell Stem Cell 12 (2013), 393–394.
12. 12 Wang, H., Yang, H., Shivalila, C.S., et al. One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering. Cell 153 (2013), 910–918.
13. 13 Niu, Y., Shen, B., Cui, Y., et al. Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos. Cell 156 (2014), 836–843.
14. 14 Liang, P., Xu, Y., Zhang, X., et al. CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes. Protein Cell 6 (2015), 363–372.
15. 15 Wu, Y., Liang, D., Wang, Y., et al. Correction of a genetic disease in mouse via use of CRISPR-Cas9. Cell Stem Cell 13 (2013), 659–662.
16. 16 Long, C., McAnally, J.R., Shelton, J.M., Mireault, A.A., Bassel-Duby, R., Olson, E.N., Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA. Science 345 (2014), 1184–1188.
17. 17 Cyranoski, D., Ethics of embryo editing divides scientists. Nature, 519, 2015, 272.
18. 18 Lanphier, E., Urnov, F., Haecker, S.E., Werner, M., Smolenski, J., Don't edit the human germ line. Nature 519 (2015), 410–411.
19. 19 Yin, H., Xue, W., Chen, S., et al. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat Biotechnol 32 (2014), 551–553.
20. 20 Ding, Q., Strong, A., Patel, K.M., et al. Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing. Circ Res 115 (2014), 488–492.
21. 21 Lin, S.R., Yang, H.C., Kuo, Y.T., et al. The CRISPR/Cas9 system facilitates clearance of the intrahepatic HBV templates in vivo. Mol Ther Nucleic Acids, 3, 2014, e186.
22. 22 Mingozzi, F., High, K.A., Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet 12 (2011), 341–355.
23. 23 Kay, M.A., State-of-the-art gene-based therapies: the road ahead. Nat Rev Genet 12 (2011), 316–328.
24. 24 Ran, F.A., Cong, L., Yan, W.X., et al. In vivo genome editing using Staphylococcus aureus Cas9. Nature 520 (2015), 186–191.
25. 25 Ran, F.A., Hsu, P.D., Lin, C.Y., et al. Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity. Cell 154 (2013), 1380–1389.
26. 26 Osborn, M.J., Gabriel, R., Webber, B.R., et al. Fanconi anemia gene editing by the CRISPR/Cas9 system. Hum Gene Ther 26 (2015), 114–126.
27. 27 Pattanayak, V., Lin, S., Guilinger, J.P., Ma, E., Doudna, J.A., Liu, D.R., High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity. Nat Biotechnol 31 (2013), 839–843.
28. 28 Fu, Y., Foden, J.A., Khayter, C., et al. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat Biotechnol 31 (2013), 822–826.
29. 29 Frock, R.L., Hu, J., Meyers, R.M., Ho, Y., Kii, E., Alt, F.W., Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases. Nat Biotechnol 33 (2014), 179–186.
30. 30 Tsai, S.Q., Zheng, Z., Nguyen, N.T., et al. GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases. Nat Biotechnol 33 (2014), 187–198.
31. 31 Kim, D., Bae, S., Park, J., et al. Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells. Nat Methods 12 (2015), 237–243 1 p following 243.
32. 32 Crosetto, N., Mitra, A., Silva, M.J., et al. Nucleotide-resolution DNA double-strand break mapping by next-generation sequencing. Nat Methods 10 (2013), 361–365.
33. 33 Cho, S.W., Kim, S., Kim, Y., et al. Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases. Genome Res 24 (2014), 132–141.
34. 34 Fu, Y., Sander, J.D., Reyon, D., Cascio, V.M., Joung, J.K., Improving CRISPR-Cas nuclease specificity using truncated guide RNAs. Nat Biotechnol 32 (2014), 279–284.
35. 35 Kim, E., Kim, S., Kim, D.H., Choi, B.S., Choi, I.Y., Kim, J.S., Precision genome engineering with programmable DNA-nicking enzymes. Genome Res 22 (2012), 1327–1333.
36. 36 Mali, P., Aach, J., Stranges, P.B., et al. CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering. Nat Biotechnol 31 (2013), 833–838.
37. 37 Tsai, S.Q., Wyvekens, N., Khayter, C., et al. Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing. Nat Biotechnol 32 (2014), 569–576.
38. 38 Guilinger, J.P., Thompson, D.B., Liu, D.R., Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification. Nat Biotechnol 32 (2014), 577–582.
39. 39 Takahashi, K., Tanabe, K., Ohnuki, M., et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131 (2007), 861–872.
40. 40 Huch, M., Gehart, H., van Boxtel, R., et al. Long-term culture of genome-stable bipotent stem cells from adult human liver. Cell 160 (2014), 299–312.
41. 41 Csaszar, E., Kirouac, D.C., Yu, M., et al. Rapid expansion of human hematopoietic stem cells by automated control of inhibitory feedback signaling. Cell Stem Cell 10 (2012), 218–229.
42. 42 Sato, T., Stange, D.E., Ferrante, M., et al. Long-term expansion of epithelial organoids from human colon, adenoma, adenocarcinoma, and Barrett's epithelium. Gastroenterology 141 (2011), 1762–1772.
43. 43 Xie, F., Ye, L., Chang, J.C., et al. Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac. Genome Res 24 (2014), 1526–1533.
44. 44 Schwank, G., Koo, B.K., Sasselli, V., et al. Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients. Cell Stem Cell 13 (2013), 653–658.
45. 45 Li, H.L., Fujimoto, N., Sasakawa, N., et al. Precise correction of the dystrophin gene in Duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9. Stem Cell Rep 4 (2014), 143–154.
46. 46 Ousterout, D.G., Kabadi, A.M., Thakore, P.I., Majoros, W.H., Reddy, T.E., Gersbach, C.A., Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy. Nat Commun, 6, 2015, 6244.
47. 47 Liao, H.K., Gu, Y., Diaz, A., et al. Use of the CRISPR/Cas9 system as an intracellular defense against HIV-1 infection in human cells. Nat Commun, 6, 2015, 6413.
48. 48 Mandal, P.K., Ferreira, L.M., Collins, R., et al. Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9. Cell Stem Cell 15 (2014), 643–652.
49. 49 Tebas, P., Stein, D., Tang, W.W., et al. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med 370 (2014), 901–910.
50. 50 Ji, J., Ng, S.H., Sharma, V., et al. Elevated coding mutation rate during the reprogramming of human somatic cells into induced pluripotent stem cells. Stem Cells 30 (2012), 435–440.
51. 51 Gore, A., Li, Z., Fung, H.L., et al. Somatic coding mutations in human induced pluripotent stem cells. Nature 471 (2011), 63–67.