Targeting hepatitis B virus cccDNA using CRISPR/Cas9

Antiviral Research

Volumn 123, Issue , 2015, Pages 188-192

  Kennedy, Edward M ^a   Kornepati, Anand V R ^a   Cullen, Bryan R ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

cccDNA; CRISPR Cas; Gene therapy; HBV; Viral persistence

Indexed keywords

CRISPR CAS SYSTEM; DRUG DEVELOPMENT; GENE THERAPY; GENETICS; HEPATITIS B VIRUS; HEPATITIS B, CHRONIC; HUMAN; PROCEDURES; TRENDS; VIROLOGY;

CRISPR-CAS SYSTEMS; DRUG DISCOVERY; GENETIC THERAPY; HEPATITIS B VIRUS; HEPATITIS B, CHRONIC; HUMANS;

EID: 84981499570     PISSN: 01663542     EISSN: 18729096     Source Type: Journal    
DOI: 10.1016/j.antiviral.2015.10.004     Document Type: Review

References (29)

1. Barrangou, R., Marraffini, L.A., CRISPR–Cas systems: prokaryotes upgrade to adaptive immunity. Mol. Cell 54 (2014), 234–244.
2. Block, T.M., Rawat, S., Brosgart, C.L., Chronic hepatitis B: a wave of new therapies on the horizon. Antiviral Res. 121 (2015), 69–81.
3. Cheng, L., Li, F., Bility, M.T., Murphy, C.M., Su, L., Modeling hepatitis B virus infection, immunopathology and therapy in mice. Antiviral Res. 121 (2015), 1–8.
4. Cong, L., Ran, F.A., Cox, D., Lin, S., Barretto, R., Habib, N., Hsu, P.D., Wu, X., Jiang, W., Marraffini, L.A., Zhang, F., Multiplex genome engineering using CRISPR/Cas systems. Science 339 (2013), 819–823.
5. Dandri, M., Lutgehetmann, M., Volz, T., Petersen, J., Small animal model systems for studying hepatitis B virus replication and pathogenesis. Semin. Liver Dis. 26 (2006), 181–191.
6. Dandri, M., Lutgehetmann, M., Petersen, J., Experimental models and therapeutic approaches for HBV. Semin. Immunopathol. 35 (2013), 7–21.
7. Dong, C., Qu, L., Wang, H., Wei, L., Dong, Y., Xiong, S., Targeting hepatitis B virus cccDNA by CRISPR/Cas9 nuclease efficiently inhibits viral replication. Antiviral Res. 118 (2015), 110–117.
8. Garneau, J.E., Dupuis, M.E., Villion, M., Romero, D.A., Barrangou, R., Boyaval, P., Fremaux, C., Horvath, P., Magadan, A.H., Moineau, S., The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA. Nature 468 (2010), 67–71.
9. Gasiunas, G., Barrangou, R., Horvath, P., Siksnys, V., Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria. Proc. Natl. Acad. Sci. U.S.A. 109 (2012), E2579–E2586.
10. Gish, R.G., Given, B.D., Lai, C.L., Locarnini, S.A., Lau, J.Y., Lewis, D.L., Schluep, T., Chronic hepatitis B: virology, natural history, current management and a glimpse at future opportunities. Antiviral Res. 121 (2015), 47–58.
11. Guo, J.T., Guo, H., Metabolism and function of hepatitis B virus cccDNA: implications for the development of cccDNA-targeting antiviral therapeutics. Antiviral Res. 122 (2015), 91–100.
12. Hsu, P.D., Lander, E.S., Zhang, F., Development and applications of CRISPR–Cas9 for genome engineering. Cell 157 (2014), 1262–1278.
13. 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.
14. Karimova, M., Beschorner, N., Dammermann, W., Chemnitz, J., Indenbirken, D., Bockmann, J.H., Grundhoff, A., Luth, S., Buchholz, F., Schulze zur Wiesch, J., Hauber, J., CRISPR/Cas9 nickase-mediated disruption of hepatitis B virus open reading frame S and X. Sci. Rep., 5, 2015, 13734.
15. Kennedy, E.M., Cullen, B.R., Bacterial CRISPR/Cas DNA endonucleases: a revolutionary technology that could dramatically impact viral research and treatment. Virology 479 (2015), 213–220.
16. Kennedy, E.M., Bassit, L.C., Mueller, H., Kornepati, A.V.R., Bogerd, H.P., Nie, T., Chatterjee, P., Javanbakht, H., Schinazi, R.F., Cullen, B.R., Suppression of hepatitis B virus DNA accumulation in chronically infected cells using a bacterial CRISPR/Cas RNA-guided DNA endonuclease. Virology 476 (2015), 196–205.
17. Larkin, J., Clayton, M., Sun, B., Perchonock, C.E., Morgan, J.L., Siracusa, L.D., Michaels, F.H., Feitelson, M.A., Hepatitis B virus transgenic mouse model of chronic liver disease. Nat. Med. 5 (1999), 907–912.
18. Lin, S.R., Yang, H.C., Kuo, Y.T., Liu, C.J., Yang, T.Y., Sung, K.C., Lin, Y.Y., Wang, H.Y., Wang, C.C., Shen, Y.C., Wu, F.Y., Kao, J.H., Chen, D.S., Chen, P.J., The CRISPR/Cas9 system facilitates clearance of the intrahepatic HBV templates in vivo. Mol. Ther. Nucleic Acids, 3, 2014, e186.
19. Lisowski, L., Dane, A.P., Chu, K., Zhang, Y., Cunningham, S.C., Wilson, E.M., Nygaard, S., Grompe, M., Alexander, I.E., Kay, M.A., Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature 506 (2014), 382–386.
20. Liu, X., Hao, R., Chen, S., Guo, D., Chen, Y., Inhibition of hepatitis B virus by the CRISPR/Cas9 system via targeting the conserved regions of the viral genome. J. Gen. Virol. 96 (2015), 2252–2261.
21. Mali, P., Yang, L., Esvelt, K.M., Aach, J., Guell, M., DiCarlo, J.E., Norville, J.E., Church, G.M., RNA-guided human genome engineering via Cas9. Science 339 (2013), 823–826.
22. Mefferd, A.L., Kornepati, A.V., Bogerd, H.P., Kennedy, E.M., Cullen, B.R., Expression of CRISPR/Cas single guide RNAs using small tRNA promoters. RNA 21 (2015), 1683–1689.
23. Ramanan, V., Shlomai, A., Cox, D.B., Schwartz, R.E., Michailidis, E., Bhatta, A., Scott, D.A., Zhang, F., Rice, C.M., Bhatia, S.N., CRISPR/Cas9 cleavage of viral DNA efficiently suppresses hepatitis B virus. Sci. Rep., 5, 2015, 10833.
24. Ran, F.A., Hsu, P.D., Lin, C.Y., Gootenberg, J.S., Konermann, S., Trevino, A.E., Scott, D.A., Inoue, A., Matoba, S., Zhang, Y., Zhang, F., Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity. Cell 154 (2013), 1380–1389.
25. Ran, F.A., Cong, L., Yan, W.X., Scott, D.A., Gootenberg, J.S., Kriz, A.J., Zetsche, B., Shalem, O., Wu, X., Makarova, K.S., Koonin, E.V., Sharp, P.A., Zhang, F., In vivo genome editing using Staphylococcus aureus Cas9. Nature 520 (2015), 186–191.
26. Sapranauskas, R., Gasiunas, G., Fremaux, C., Barrangou, R., Horvath, P., Siksnys, V., The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli. Nucleic Acids Res. 39 (2011), 9275–9282.
27. Seeger, C., Sohn, J.A., Targeting hepatitis B virus with CRISPR/Cas9. Mol. Ther. Nucleic Acids, 3, 2014, e216.
28. Wang, J., Xu, Z.W., Liu, S., Zhang, R.Y., Ding, S.L., Xie, X.M., Long, L., Chen, X.M., Zhuang, H., Lu, F.M., Dual gRNAs guided CRISPR/Cas9 system inhibits hepatitis B virus replication. World J. Gastroenterol. 21 (2015), 9554–9565.
29. Zhen, S., Hua, L., Liu, Y.H., Gao, L.C., Fu, J., Wan, D.Y., Dong, L.H., Song, H.F., Gao, X., Harnessing the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated Cas9 system to disrupt the hepatitis B virus. Gene Ther. 22 (2015), 404–412.