Zinc finger nuclease: a new approach for excising HIV-1 proviral DNA from infected human T cells

详细信息    查看全文

• 作者：Xiying Qu ; Pengfei Wang ; Donglin Ding ; Xiaohui Wang…
• 关键词：Human immunodeficiency virus 1 ; Zinc ; finger nucleases ; Long terminal repeat
• 刊名：Molecular Biology Reports
• 出版年：2014
• 出版时间：September 2014
• 年：2014
• 卷：41
• 期：9
• 页码：5819-5827
• 全文大小：1,970 KB
• 参考文献：1. Richman DD, Margolis DM, Delaney M, Greene WC, Hazuda D, Pomerantz RJ (2009) The challenge of finding a cure for HIV infection. Science 323:1304-307 CrossRef
  2. Katlama C, Deeks SG, Autran B, Martinez-Picado J, van Lunzen J, Rouzioux C, Miller M, Vella S, Schmitz JE, Ahlers J, Richman DD, Sekaly RP (2013) Barriers to a cure for HIV: new ways to target and eradicate HIV-1 reservoirs. Lancet 381:2109-117 CrossRef
  3. Marcellin F, Spire B, Carrieri MP, Roux P (2013) Assessing adherence to antiretroviral therapy in randomized HIV clinical trials: a review of currently used methods. Expert Rev Anti Infect Ther 11:239-50 CrossRef
  4. Martinez-Colom A, Lasarte S, Fernandez-Pineda A, Relloso M, Munoz-Fernandez MA (2013) A new chimeric protein represses HIV-1 LTR-mediated expression by DNA methylase. Antivir Res 98:394-00 CrossRef
  5. Nisole S, Sa?b A (2004) Early steps of retrovirus replicative cycle. Retrovirology 1:9 CrossRef
  6. Chun T-W, Stuyver L, Mizell SB, Ehler LA, Mican JAM, Baseler M, Lloyd AL, Nowak MA, Fauci AS (1997) Presence of an inducible HIV-1 latent reservoir during highly active antiretroviral therapy. Proc Natl Acad Sci 94:13193-3197 CrossRef
  7. Finzi D, Hermankova M, Pierson T, Carruth LM, Buck C, Chaisson RE, Quinn TC, Chadwick K, Margolick J, Brookmeyer R (1997) Identification of a reservoir for HIV-1 in patients on highly active antiretroviral therapy. Science 278:1295-300 CrossRef
  8. Trono D, Van Lint C, Rouzioux C, Verdin E, Barre′-Sinoussi F, Chun T-W, Chomont N (2010) HIV persistence and the prospect of long-term drug-free remissions for HIV-infected individuals. Science 329:174-80 CrossRef
  9. Taube R, Peterlin M (2013) Lost in transcription: molecular mechanisms that control HIV latency. Viruses 5:902-27 CrossRef
  10. Mussolino C, Morbitzer R, Lutge F, Dannemann N, Lahaye T, Cathomen T (2011) A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. Nucleic Acids Res 39:9283-293 CrossRef
  11. Schiffer JT, Aubert M, Weber ND, Mintzer E, Stone D, Jerome KR (2012) Targeted DNA mutagenesis for the cure of chronic viral infections. J Virol 86:8920-936 CrossRef
  12. Ebina H, Misawa N, Kanemura Y, Koyanagi Y (2013) Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus. Sci Rep 3:2510 CrossRef
  13. Sarkar I, Hauber I, Hauber J, Buchholz F (2007) HIV-1 proviral DNA excision using an evolved recombinase. Science 316:1912-915 CrossRef
  14. Aubert M, Ryu BY, Banks L, Rawlings DJ, Scharenberg AM, Jerome KR (2011) Successful targeting and disruption of an integrated reporter lentivirus using the engineered homing endonuclease Y2 I-AniI. PLoS One 6:e16825 CrossRef
  15. Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD (2010) Genome editing with engineered zinc finger nucleases. Nat Rev Genet 11:636-46 CrossRef
  16. Bibikova M, Beumer K, Trautman JK, Carroll D (2003) Enhancing gene targeting with designed zinc finger nucleases. Science 300:764 CrossRef
  17. Lee HJ, Kim E, Kim J-S (2010) Targeted chromosomal deletions in human cells using zinc finger nucleases. Genome Res 20:81-9 CrossRef
  18. Urnov FD, Miller JC, Lee Y-L, Beausejour CM, Rock JM, Augustus S, Jamieson AC, Porteus MH, Gregory PD, Holmes MC (2005) Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435:646-51 CrossRef
  19. Perez EE, Wang J, Miller JC, Jouvenot Y, Kim KA, Liu O, Wang N, Lee G, Bartsevich VV, Lee Y-L (2008) Establishment of HIV-1 resistance in CD4?+?T cells by genome editing using zinc-finger nucleases. Nat Biotechnol 26:808-16 CrossRef
  20. Holt N, Wang J, Kim K, Friedman G, Wang X, Taupin V, Crooks GM, Kohn DB, Gregory PD, Holmes MC (2010) Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol 28:839-47 CrossRef
  21. Hockemeyer D, Soldner F, Beard C, Gao Q, Mitalipova M, DeKelver RC, Katibah GE, Amora R, Boydston EA, Zeitler B (2009) Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 27:851-57 CrossRef
  22. Doyon Y, McCammon JM, Miller JC, Faraji F, Ngo C, Katibah GE, Amora R, Hocking TD, Zhang L, Rebar EJ (2008) Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases. Nat Biotechnol 26:702-08 CrossRef
  23. Li H, Haurigot V, Doyon Y, Li T, Wong SY, Bhagwat AS, Malani N, Anguela XM, Sharma R, Ivanciu L (2011) In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature 475:217-21 CrossRef
  24. Yang D, Yang H, Li W, Zhao B, Ouyang Z, Liu Z, Zhao Y, Fan N, Song J, Tian J (2011) Generation of PPARγ mono-allelic knockout pigs via zinc-finger nucleases and nuclear transfer cloning. Cell Res 21:979-82 CrossRef
  25. Cui X, Ji D, Fisher DA, Wu Y, Briner DM, Weinstein EJ (2010) Targeted integration in rat and mouse embryos with zinc-finger nucleases. Nat Biotechnol 29:64-7 CrossRef
  26. Geurts AM, Cost GJ, Freyvert Y, Zeitler B, Miller JC, Choi VM, Jenkins SS, Wood A, Cui X, Meng X (2009) Knockout rats via embryo microinjection of zinc-finger nucleases. Science 325:433 CrossRef
  27. Ochiai H, Sakamoto N, Fujita K, Nishikawa M, Suzuki K-i, Matsuura S, Miyamoto T, Sakuma T, Shibata T, Yamamoto T (2012) Zinc-finger nuclease-mediated targeted insertion of reporter genes for quantitative imaging of gene expression in sea urchin embryos. Proc Natl Acad Sci 109:10915-0920 CrossRef
  28. Merlin C, Beaver LE, Taylor OR, Wolfe SA, Reppert SM (2013) Efficient targeted mutagenesis in the monarch butterfly using zinc-finger nucleases. Genome Res 23:159-68 CrossRef
  29. Segal DJ, Gon?alves J, Eberhardy S, Swan CH, Torbett BE, Li X, Barbas CF (2004) Attenuation of HIV-1 replication in primary human cells with a designed zinc finger transcription factor. J Biol Chem 279:14509-4519 CrossRef
  30. Eberhardy SR, Goncalves J, Coelho S, Segal DJ, Berkhout B, Barbas CF (2006) Inhibition of human immunodeficiency virus type 1 replication with artificial transcription factors targeting the highly conserved primer-binding site. J Virol 80:2873-883 CrossRef
  31. Sakkhachornphop S, Jiranusornkul S, Kodchakorn K, Nangola S, Sirisanthana T, Tayapiwatana C (2009) Designed zinc finger protein interacting with the HIV-1 integrase recognition sequence at 2-LTR-circle junctions. Protein Sci 18:2219-230 CrossRef
  32. Sakkhachornphop S, Barbas CF III, Keawvichit R, Wongworapat K, Tayapiwatana C (2012) Zinc finger protein designed to target 2-long terminal repeat junctions interferes with human immunodeficiency virus integration. Hum Gene Ther 23:932-42 CrossRef
  33. Qu X, Wang P, Ding D, Li L, Wang H, Ma L, Zhou X, Liu S, Lin S, Wang X, Zhang G, Liu L, Wang J, Zhang F, Lu D, Zhu H (2013) Zinc-finger-nucleases mediate specific and efficient excision of HIV-1 proviral DNA from infected and latently infected human T cells. Nucleic Acids Res 41:7771-782 CrossRef
  34. Jordan A, Bisgrove D, Verdin E (2003) HIV reproducibly establishes a latent infection after acute infection of T cells in vitro. EMBO J 22:1868-877 CrossRef
  35. Tanaka A, Takeda S, Kariya R, Matsuda K, Urano E, Okada S, Komano J (2013) A novel therapeutic molecule against HTLV-1 infection targeting provirus. Leukemia 27:1621-627 CrossRef
  
• 作者单位：Xiying Qu (1)
  Pengfei Wang (1)
  Donglin Ding (1)
  Xiaohui Wang (1)
  Gongmin Zhang (1)
  Xin Zhou (1)
  Lin Liu (1)
  Xiaoli Zhu (1)
  Hanxian Zeng (1)
  Huanzhang Zhu (1)
  
  1. State Key Laboratory of Genetic Engineering, School of Life Sciences, Institute of Genetics, Fudan University, Shanghai, 200433, China
  
• ISSN：1573-4978

文摘

A major reason that Acquired Immune Deficiency Syndrome (AIDS) cannot be completely cured is the human immunodeficiency virus 1 (HIV-1) provirus integrated into the human genome. Though existing therapies can inhibit replication of HIV-1, they cannot eradicate it. A molecular therapy gains popularity due to its specifically targeting to HIV-1 infected cells and effectively removing the HIV-1, regardless of viral genes being active or dormant. Now, we propose a new method which can excellently delete the HIV provirus from the infected human T cell genome. First, we designed zinc-finger nucleases (ZFNs) that target a sequence within the long terminal repeat (LTR) U3 region that is highly conserved in whole clade. Then, we screened out one pair of ZFN and named it as ZFN-U3. We discovered that ZFN-U3 can exactly target and eliminate the full-length HIV-1 proviral DNA after the infected human cell lines treated with it, and the frequency of its excision was about 30?% without cytotoxicity. These results prove that ZFN-U3 can effciently excise integrated HIV-1 from the human genome in infected cells. This method to delete full length HIV-1 in human genome can therefore provide a novel approach to cure HIV-infected individuals in the future.