Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus

Molecular Therapy

Volumn 21, Issue 9, 2013, Pages 1705-1717

  Joglekar, Alok V ^a,b   Hollis, Roger P ^a,b   Kuftinec, Gabriela ^a,b   Senadheera, Shantha ^a,b   Chan, Rebecca ^a,b   Kohn, Donald B ^a,b  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; INTEGRASE DEFECTIVE LENTIVIRAL VECTOR; MESSENGER RNA; UNCLASSIFIED DRUG; VIRUS VECTOR; ZINC FINGER NUCLEASE;

ADENOSINE DEAMINASE GENE; ARTICLE; CELL STRAIN K 562; CONTROLLED STUDY; DNA MODIFICATION; DNA TEMPLATE; DRUG CYTOTOXICITY; DRUG EFFICACY; GENE EDITING; GENE EXPRESSION; GENETIC ENGINEERING; GENETIC RECOMBINATION; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; NONHUMAN; TRANSIENT TRANSFECTION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS GENOME;

ADENOSINE DEAMINASE; ENDONUCLEASES; GENETIC LOCI; GENETIC VECTORS; HUMANS; INTEGRASES; K562 CELLS; LENTIVIRUS; TRANSDUCTION, GENETIC; VIRUS INTEGRATION; ZINC FINGERS;

EID: 84883743436     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.106     Document Type: Article

References (35)

1. Urnov, FD, Miller, JC, Lee, YL, Beausejour, CM, Rock, JM, Augustus, S et al. (2005). Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435: 646-651. (Pubitemid 40825505)
2. Klug, A (2010). The discovery of zinc fingers and their applications in gene regulation and genome manipulation. Annu Rev Biochem 79: 213-231.
3. Urnov, FD, Rebar, EJ, Holmes, MC, Zhang, HS and Gregory, PD (2010). Genome editing with engineered zinc finger nucleases. Nat Rev Genet 11: 636-646.
4. Carroll, D (2008). Progress and prospects: zinc-finger nucleases as gene therapy agents. Gene Ther 15: 1463-1468.
5. Zou, J, Sweeney, CL, Chou, BK, Choi, U, Pan, J, Wang, H et al. (2011). Oxidasedeficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting. Blood 117: 5561-5572.
6. Holt, N, Wang, J, Kim, K, Friedman, G, Wang, X, Taupin, V et al. (2010). Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol 28: 839-847.
7. Perez, EE, Wang, J, Miller, JC, Jouvenot, Y, Kim, KA, Liu, O et al. (2008). Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases. Nat Biotechnol 26: 808-816. (Pubitemid 351961450)
8. Hockemeyer, D, Soldner, F, Beard, C, Gao, Q, Mitalipova, M, DeKelver, RC et al. (2009). Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 27: 851-857.
9. Li, H, Haurigot, V, Doyon, Y, Li, T, Wong, SY, Bhagwat, AS et al. (2011). In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature 475: 217-221.
10. Lombardo, A, Genovese, P, Beausejour, CM, Colleoni, S, Lee, YL, Kim, KA et al. (2007). Gene editing in human stem cells using zinc finger nucleases and integrasedefective lentiviral vector delivery. Nat Biotechnol 25: 1298-1306. (Pubitemid 350076515)
11. Provasi, E, Genovese, P, Lombardo, A, Magnani, Z, Liu, PQ, Reik, A et al. (2012). Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer. Nat Med 18: 807-815.
12. Asokan, A, Schaffer, DV and Samulski, RJ (2012). The AAV vector toolkit: poised at the clinical crossroads. Mol Ther 20: 699-708.
13. Hofmann, A, Kessler, B, Ewerling, S, Kabermann, A, Brem, G, Wolf, E et al. (2006). Epigenetic regulation of lentiviral transgene vectors in a large animal model. Mol Ther 13: 59-66. (Pubitemid 41763483)
14. Naldini, L, Bller, U, Gage, FH, Trono, D and Verma, IM (1996). Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc Natl Acad Sci USA 93: 11382-11388. (Pubitemid 26347130)
15. Vargas, J Jr, Gusella, GL, Najfeld, V, Klotman, ME and Cara, A (2004). Novel integrase-defective lentiviral episomal vectors for gene transfer. Hum Gene Ther 15: 361-372. (Pubitemid 38469778)
16. Nightingale, SJ, Hollis, RP, Pepper, KA, Petersen, D, Yu, XJ, Yang, C et al. (2006). Transient gene expression by nonintegrating lentiviral vectors. Mol Ther 13: 1121-1132. (Pubitemid 43796157)
17. Cornu, TI and Cathomen, T (2007). Targeted genome modifications using integrasedeficient lentiviral vectors. Mol Ther 15: 2107-2113. (Pubitemid 350148926)
18. Doyon, Y, Vo, TD, Mendel, MC, Greenberg, SG, Wang, J, Xia, DF et al. (2011). Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures. Nat Methods 8: 74-79.
19. Guschin, DY, Waite, AJ, Katibah, GE, Miller, JC, Holmes, MC and Rebar, EJ (2010). A rapid and general assay for monitoring endogenous gene modification. Methods Mol Biol 649: 247-256.
20. Zufferey, R, Donello, JE, Trono, D and Hope, TJ (1999). Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J Virol 73: 2886-2892. (Pubitemid 29135675)
21. Zychlinski, D, Schambach, A, Modlich, U, Maetzig, T, Meyer, J, Grassman, E et al. (2008). Physiological promoters reduce the genotoxic risk of integrating gene vectors. Mol Ther 16: 718-725. (Pubitemid 351426173)
22. Kim, JH, Lee, SR, Li, LH, Park, HJ, Park, JH, Lee, KY et al. (2011). High cleavage efficiency of a 2A peptide derived from porcine teschovirus-1 in human cell lines, zebrafish and mice. PLoS ONE 6: e18556.
23. Cooper, AR, Patel, S, Senadheera, S, Plath, K, Kohn, DB and Hollis, RP (2011). Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration. J Virol Methods 177: 1-9.
24. Maeder, ML, Thibodeau-Beganny, S, Sander, JD, Voytas, DF and Joung, JK (2009). Oligomerized pool engineering (OPEN): an 'open-source' protocol for making customized zinc-finger arrays. Nat Protoc 4: 1471-1501.
25. Wright, DA, Thibodeau-Beganny, S, Sander, JD, Winfrey, RJ, Hirsh, AS, Eichtinger, M et al. (2006). Standardized reagents and protocols for engineering zinc finger nucleases by modular assembly. Nat Protoc 1: 1637-1652.
26. Papadakis, ED, Nicklin, SA, Baker, AH and White, SJ (2004). Promoters and control elements: designing expression cassettes for gene therapy. Curr Gene Ther 4: 89-113. (Pubitemid 38854948)
27. Robbins, PB, Yu, XJ, Skelton, DM, Pepper, KA, Wasserman, RM, Zhu, L et al. (1997). Increased probability of expression from modified retroviral vectors in embryonal stem cells and embryonal carcinoma cells. J Virol 71: 9466-9474. (Pubitemid 27492391)
28. Reiser, J, Harmison, G, Kluepfel-Stahl, S, Brady, RO, Karlsson, S and Schubert, M (1996). Transduction of nondividing cells using pseudotyped defective high-titer HIV type 1 particles. Proc Natl Acad Sci USA 93: 15266-15271.
29. Cartier, N, Hacein-Bey-Abina, S, Bartholomae, CC, Veres, G, Schmidt, M, Kutschera, I et al. (2009). Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326: 818-823.
30. Leath, A and Cornetta, K (2012). Developing novel lentiviral vectors into clinical products. Meth Enzymol 507: 89-108.
31. Basu, VP, Song, M, Gao, L, Rigby, ST, Hanson, MN and Bambara, RA (2008). Strand transfer events during HIV-1 reverse transcription. Virus Res 134: 19-38.
32. Fuentes, GM, Palaniappan, C, Fay, PJ and Bambara, RA (1996). Strand displacement synthesis in the central polypurine tract region of HIV-1 promotes DNA to DNA strand transfer recombination. J Biol Chem 271: 29605-29611. (Pubitemid 26389582)
33. Schambach, A, Bohne, J, Chandra, S, Will, E, Margison, GP, Williams, DA et al. (2006). Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. Mol Ther 13: 391-400. (Pubitemid 43056905)
34. Leuci, V, Mesiano, G, Gammaitoni, L, Cammarata, C, Capellero, S, Todorovic, M et al. (2011). Transient proteasome inhibition as a strategy to enhance lentiviral transduction of hematopoietic CD34(+) cells and T lymphocytes: implications for the use of low viral doses and large-size vectors. J Biotechnol 156: 218-226.
35. Pelascini, LP, Janssen, JM and Gonlves, MA (2013). Histone deacetylase inhibition activates transgene expression from integration-defective lentiviral vectors in dividing and non-dividing cells. Hum Gene Ther 24: 78-96.