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25 - Gene Silencing, Disruption and Latency Reactivation with RNA-based and Gene Editing CRISPR/Cas, ZFN and TALEN Technologies for HIV-1/AIDS Therapies

from Part V - Genome Editing in Disease Biology

Published online by Cambridge University Press:  30 July 2018

Krishnarao Appasani
Affiliation:
GeneExpression Systems, Inc.
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Summary

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Type
Chapter
Information
Genome Editing and Engineering
From TALENs, ZFNs and CRISPRs to Molecular Surgery
, pp. 389 - 400
Publisher: Cambridge University Press
Print publication year: 2018

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References

Akkina, R. 2013. New generation humanized mice for virus research: comparative aspects and future prospects. Virology 435: 1428.CrossRefGoogle ScholarPubMed
Anderson, J, Li, MJ, Palmer, B, et al. 2007. Safety and efficacy of a lentiviral vector containing three anti-HIV genes – CCR5 ribozyme, tat-rev siRNA, and TAR decoy – in SCID-hu mouse-derived T cells. Mol Ther 15: 11821188.CrossRefGoogle ScholarPubMed
Berkhout, B. 2004. RNA interference as an antiviral approach: targeting HIV-1. Curr Opin Mol Ther 6: 141145.Google ScholarPubMed
Bobbin, ML, Burnett, JC, Rossi, JJ. 2015. RNA interference approaches for treatment of HIV-1 infection. Genome Med 7: 50.CrossRefGoogle ScholarPubMed
Choi, JG, Dang, Y, Abraham, S, et al. 2016. Lentivirus pre-packed with Cas9 protein for safer gene editing. Gene Ther 23: 627633.CrossRefGoogle ScholarPubMed
Hou, P, Chen, S, Wang, S, et al. 2015. Genome editing of CXCR4 by CRISPR/cas9 confers cells resistant to HIV-1 infection. Sci Rep 5: 15577.CrossRefGoogle ScholarPubMed
Kaminski, R, Bella, R, Yin, C, et al. 2016a. Excision of HIV-1 DNA by gene editing: a proof-of-concept in vivo study. Gene Ther 23: 696.CrossRefGoogle ScholarPubMed
Kaminski, R, Chen, Y, Salkind, J, et al. 2016b. Negative feedback regulation of HIV-1 by gene editing strategy. Sci Rep 6: 31527.CrossRefGoogle ScholarPubMed
Keefe, AD, Pai, S, Ellington, A. 2010. Aptamers as therapeutics. Nat Rev Drug Disc 9: 537550.CrossRefGoogle ScholarPubMed
Kim, SS, Peer, D, Kumar, P, et al. 2010. RNAi-mediated CCR5 silencing by LFA-1-targeted nanoparticles prevents HIV infection in BLT mice. Mol Ther 18: 370376.CrossRefGoogle ScholarPubMed
Kumar, P, Ban, HS, Kim, SS, et al. 2008. T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice. Cell 134: 577586.CrossRefGoogle ScholarPubMed
Li, L, Krymskaya, L, Wang, J, et al. 2013. Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases. Mol Ther 21: 12591269.CrossRefGoogle ScholarPubMed
Liang, C, Wainberg, MA, Das, AT, Berkhout, B. 2016. CRISPR/Cas9: a double-edged sword when used to combat HIV infection. Retrovirology 13: 37.CrossRefGoogle ScholarPubMed
Limsirichai, P, Gaj, T, Schaffer, DV. 2016. CRISPR-mediated activation of latent HIV-1 expression. Mol Ther 24: 499507.CrossRefGoogle ScholarPubMed
Lin, A, Klase, Z. 2016. A CRISPR approach for reactivating latent HIV-1. Mol Ther 24: 416418.CrossRefGoogle ScholarPubMed
Maier, DA, Brennan, AL, Jiang, S, et al. 2013. Efficient clinical scale gene modification via zinc finger nuclease-targeted disruption of the HIV co-receptor CCR5. Hum Gene Ther 24: 245258.CrossRefGoogle ScholarPubMed
Malecova, B, Morris, KV. 2010. Transcriptional gene silencing through epigenetic changes mediated by non-coding RNAs. Curr Opin Mol Ther 12: 214222.Google ScholarPubMed
Mussolino, C, Morbitzer, R, Lutge, F, et al. 2011. A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. Nucleic Acids Res 39: 92839293.CrossRefGoogle ScholarPubMed
Neff, CP, Zhou, J, Remling, L, et al. 2011. An aptamer-siRNA chimera suppresses HIV-1 viral loads and protects from helper CD4(+) T cell decline in humanized mice. Sci Transl Med 3: 66ra6.CrossRefGoogle ScholarPubMed
Perez, EE, Wang, J, Miller, JC, et al. 2008. Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases. Nat Biotechnol 26: 808816.CrossRefGoogle ScholarPubMed
Saayman, SM, Lazar, DC, Scott, TA, et al. 2016. Potent and targeted activation of latent HIV-1 using the CRISPR/dCas9 activator complex. Mol Ther 24: 488498.CrossRefGoogle ScholarPubMed
Saayman, S, Roberts, TC, Morris, KV, Weinberg, MS. 2015. HIV latency and the noncoding RNA therapeutic landscape. Adv Exp Med Biol 848: 169189.CrossRefGoogle ScholarPubMed
Tebas, P, Stein, D, Tang, WW, et al. 2014. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med 370: 901910.CrossRefGoogle ScholarPubMed
Thiel, KW, Giangrande, PH. 2010. Intracellular delivery of RNA-based therapeutics using aptamers. Ther Del 1: 849861.CrossRefGoogle ScholarPubMed
Ueda, S, Ebina, H, Kanemura, Y, Misawa, N, Koyanagi, Y. 2016. Anti-HIV-1 potency of the CRISPR/Cas9 system insufficient to fully inhibit viral replication. Microbiol Immunol 60: 483496.CrossRefGoogle ScholarPubMed
Wang, G, Zhao, N, Berkhout, B, Das, AT. 2016a. CRISPR-Cas9 can inhibit HIV-1 replication but NHEJ repair facilitates virus escape. Mol Ther 24: 522526.CrossRefGoogle ScholarPubMed
Wang, Z, Pan, Q, Gendron, P, et al. 2016b. CRISPR/Cas9-derived mutations both inhibit HIV-1 replication and accelerate viral escape. Cell Rep 15: 481489.CrossRefGoogle ScholarPubMed
Whatley, AS, Ditzler, MA, Lange, MJ, et al. 2013. Potent inhibition of HIV-1 reverse transcriptase and replication by nonpseudoknot, “UCAA-motif” RNA aptamers. Mol Ther Nucleic Acids 2: e71.CrossRefGoogle ScholarPubMed
Wheeler, LA, Vrbanac, V, Trifonova, R, et al. 2013. Durable knockdown and protection from HIV transmission in humanized mice treated with gel-formulated CD4 aptamer-siRNA chimeras. Mol Ther 21: 13781389.CrossRefGoogle ScholarPubMed
Ye, L, Wang, J, Beyer, AI, et al. 2014. Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Delta32 mutation confers resistance to HIV infection. Proc Natl Acad Sci USA 111: 95919596.CrossRefGoogle Scholar
Yuan, J, Wang, J, Crain, K, et al. 2012. Zinc-finger nuclease editing of human cxcr4 promotes HIV-1 CD4(+) T cell resistance and enrichment. Mol Ther 20: 849859.CrossRefGoogle ScholarPubMed
Zhou, J, Satheesan, S, Li, H, et al. 2015. Cell-specific RNA aptamer against human CCR5 specifically targets HIV-1 susceptible cells and inhibits HIV-1 infectivity. Chem Biol 22: 379390.CrossRefGoogle ScholarPubMed
Zhou, J, Swiderski, P, Li, H, et al. 2009. Selection, characterization and application of new RNA HIV gp 120 aptamers for facile delivery of Dicer substrate siRNAs into HIV infected cells. Nucleic Acids Res 37: 30943109.CrossRefGoogle ScholarPubMed
Zhu, W, Lei, R, Le Duff, Y, et al. 2015. The CRISPR/Cas9 system inactivates latent HIV-1 proviral DNA. Retrovirology 12: 22.CrossRefGoogle ScholarPubMed

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