Novel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleases

Scientific Reports

Volumn 4, Issue , 2014, Pages

  Mock, Ulrike ^a   Riecken, Kristoffer ^a   Berdien, Belinda ^a   Qasim, Waseem ^b   Chan, Emma ^b   Cathomen, Toni ^c   Fehse, Boris ^a  

^a UNIVERSITY MEDICAL CENTER HAMBURG EPPENDORF   (Germany)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c UNIVERSITY OF FREIBURG   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

CHEMOKINE RECEPTOR CCR5; ENDONUCLEASE; MESSENGER RNA; RNA DIRECTED DNA POLYMERASE;

DRUG DELIVERY SYSTEM; GENE EXPRESSION REGULATION; GENE TRANSFER; GENE VECTOR; GENETIC ENGINEERING; GENETIC TRANSDUCTION; GENETICS; HEK293 CELL LINE; HUMAN; HUMAN GENOME; LENTIVIRINAE; METABOLISM; MUTATION;

DRUG DELIVERY SYSTEMS; ENDONUCLEASES; GENE EXPRESSION REGULATION; GENE TRANSFER TECHNIQUES; GENETIC ENGINEERING; GENETIC VECTORS; GENOME, HUMAN; HEK293 CELLS; HIV REVERSE TRANSCRIPTASE; HUMANS; LENTIVIRUS; MUTATION; RECEPTORS, CCR5; RNA, MESSENGER; TRANSDUCTION, GENETIC;

EID: 84923381487     PISSN: None     EISSN: 20452322     Source Type: Journal    
DOI: 10.1038/srep06409     Document Type: Article

References (53)

1. 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, 636-46 (2010).
2. Sander, J. D. & Joung, J. K. CRISPR-Cas systems for editing, regulating and targeting genomes. Nat. Biotechnol. 32, 347-55 (2014).
3. Joung, J. K. & Sander, J. D. TALENs: a widely applicable technology for targeted genome editing. Nat. Rev. Mol. Cell Biol. 14, 49-55 (2012).
4. Cong, L. et al.Multiplex genome engineering using CRISPR/Cas systems. Science 339, 819-23 (2013).
5. Cradick, T. J., Fine, E. J., Antico, C. J. & Bao, G. CRISPR/Cas9 systems targeting bglobin and CCR5 genes have substantial off-target activity. Nucleic Acids Res. 41, 9584-92 (2013).
6. Fu, Y. et al. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat. Biotechnol. 31, 822-826 (2013).
7. Mussolino, C. et al. TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity. Nucleic Acids Res. 42, 6762-73 (2014).
8. Chuah, M. K. & Vandendriessche, T. Optimizing delivery and expression of designer nucleases for genome engineering. Hum. Gene Ther. Methods 24, 329-32 (2013).
9. Zhang, F., Wen, Y. & Guo, X. CRISPR/Cas9 for genome editing: progress, implications and challenges. Hum. Mol. Genet. (2014) doi:10.1093/hmg/ddu125.
10. Anderson, W. F. Human gene therapy. Nature 392, 25-30 (1998).
11. Weber, K., Bartsch, U., Stocking, C. & Fehse, B. A multicolor panel of novel lentiviral "gene ontology" (LeGO) vectors for functional gene analysis. Mol. Ther. 16, 698-706 (2008).
12. Holkers, M. et al. Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells. Nucleic Acids Res. 41, e63 (2012).
13. Holkers, M., Cathomen, T. & Goncąlves, M. A. F. V. Construction and characterization of adenoviral vectors for the delivery of TALENs into human cells. Methods in press, (2014).
14. He, C. et al. Lentiviral protein delivery of meganucleases in human cells mediates gene targeting and alleviates toxicity. Gene Ther. 21, 759-66 (2014).
15. Stephen, S. L. et al. Chromosomal integration of adenoviral vector DNA in vivo. J. Virol. 84, 9987-9994 (2010).
16. Mussolino, C. et al. A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. Nucleic Acids Res. 39, 9283-9293 (2011).
17. An, W. & Telesnitsky, A. Frequency of direct repeat deletion in a human immunodeficiency virus type 1 vector during reverse transcription in human cells. Virology 286, 475-482 (2001).
18. Szymczak, A. L. et al. Correction of multi-gene deficiency in vivo using a single "self-cleaving" 2A peptide-based retroviral vector. Nat. Biotechnol. 22, 589-594 (2004).
19. Zhou, Q. et al. T-cell receptor gene transfer exclusively to human CD81 cells enhances tumor cell killing. Blood 120, 4334-42 (2012).
20. Perez, E. E. et al. Establishment of HIV-1 resistance in CD41 T cells by genome editing using zinc-finger nucleases. Nat. Biotechnol. 26, 808-816 (2008).
21. Miller, J. C. et al. An improved zinc-finger nuclease architecture for highly specific genome editing. Nat. Biotechnol. 25, 778-785 (2007).
22. Hu, W. S. & Temin, H. M. Retroviral recombination and reverse transcription. Science 250, 1227-1233 (1990).
23. Stuhlmann, H. & Berg, P. Homologous recombination of copackaged retrovirus RNAs during reverse transcription. J. Virol. 66, 2378-2388 (1992).
24. Wakefield, J. K., Jablonski, S. A. & Morrow, C. D. In vitro enzymatic activity of human immunodeficiency virus type 1 reverse transcriptase mutants in the highly conserved YMDD amino acid motif correlates with the infectious potential of the proviral genome. J. Virol. 66, 6806-6812 (1992).
25. Argos, P. A sequence motif in many polymerases. Nucleic Acids Res. 16, 9909-9916 (1988).
26. Halter, M., Tona, A., Bhadriraju, K., Plant, A. L. & Elliott, J. T. Automated live cell imaging of green fluorescent protein degradation in individual fibroblasts. Cytom. Part A 71A, 827-34 (2007).
27. Tell, G., Di Piazza, M., Kamocka, M. M. & Vascotto, C. Combining RNAi and in vivo confocal microscopy analysis of the photoconvertible fluorescent protein Dendra2 to study a DNA repair protein. Biotechniques 55, 198-203 (2013).
28. Galla, M., Will, E., Kraunus, J., Chen, L. & Baum, C. Retroviral pseudotransduction for targeted cell manipulation. Mol. Cell 16, 309-315 (2004).
29. Bobis-Wozowicz, S. et al. Non-integrating gamma-retroviral vectors as a versatile tool for transient zinc-finger nuclease delivery. Sci. Rep. 4, 4656 (2014).
30. Bunnell, B. A., Muul, L. M., Donahue, R. E., Blaese, R. M. & Morgan, R. A. Highefficiency retroviral-mediated gene transfer into human and nonhuman primate peripheral blood lymphocytes. Proc. Natl. Acad. Sci. U. S. A. 92, 7739-7743 (1995).
31. Doyon, Y. et al. Transient cold shock enhances zinc-finger nuclease-mediated gene disruption. Nat. Methods 7, 459-60 (2010).
32. Schambach, A., Galla, M., Maetzig, T., Loew, R. & Baum, C. Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors. Mol. Ther. 15, 1167-1173 (2007).
33. Hager, S., Frame, F. M., Collins, A. T., Burns, J. E. & Maitland, N. J. An internal polyadenylation signal substantially increases expression levels of lentivirusdelivered transgenes but has the potential to reduce viral titer in a promoterdependent manner. Hum. Gene Ther. 19, 840-850 (2008).
34. Zufferey, R., Donello, J. E., Trono, D. & Hope, T. J. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73, 2886-2892 (1999).
35. Schambach, A. et al. Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors. Mol. Ther. 2, 435-45 (2000).
36. Berdien, B., Mock, U., Atanackovic, D. & Fehse, B. TALEN-mediated editing of endogenous T-cell receptors facilitates efficient reprogramming of T lymphocytes by lentiviral gene transfer. Gene Ther. 21, 539-548 (2014).
37. Tebas, P. et al.Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N. Engl. J. Med. 370, 901-10 (2014).
38. Huang, X. et al. Unexpectedly high copy number of random integration but low frequency of persistent expression of the Sleeping Beauty transposase after trans delivery in primary human T cells. Hum. Gene Ther. 21, 1577-90 (2010).
39. Miller, D. G., Rutledge, E. A. & Russell, D. W. Chromosomal effects of adenoassociated virus vector integration. Nat. Genet. 30, 147-148 (2002).
40. Liu, J., Gaj, T., Patterson, J. T., Sirk, S. J. & Barbas III, C. F. Cell-Penetrating Peptide-Mediated Delivery of TALEN Proteins via Bioconjugation for Genome Engineering. PLoS One 9, e85755 (2014).
41. Ginn, S. L., Alexander, I. E., Edelstein, M. L., Abedi, M. R. & Wixon, J. Gene therapy clinical trials worldwide to 2012-an update. J. Gene Med. 15, 65-77 (2013).
42. Anliker, B. et al. Specific gene transfer to neurons, endothelial cells and hematopoietic progenitors with lentiviral vectors. Nat. Methods 7, 929-35 (2010).
43. Li, Z. et al.Murine leukemia induced by retroviral gene marking. Science 296, 497 (2002).
44. Hacein-Bey-Abina, S. et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302, 415-9 (2003).
45. Yáñez-Munõz, R. J. et al. Effective gene therapy with nonintegrating lentiviral vectors. Nat. Med. 12, 348-853 (2006).
46. Sanjana, N. E. et al. A transcription activator-like effector toolbox for genome engineering. Nat. Protoc. 7, 171-192 (2012).
47. Kaminski, A., Howell, M. T. & Jackson, R. J. Initiation of encephalomyocarditis virus RNA translation: the authentic initiation site is not selected by a scanning mechanism. EMBO J. 9, 3753-9 (1990).
48. Mock, U., Thiele, R., Uhde, A., Fehse, B. & Horn, S. Efficient lentiviral transduction and transgene expression in primary human B cells. Hum. Gene Ther. Methods 23, 408-415 (2012).
49. Dull, T. et al. A third-generation lentivirus vector with a conditional packaging system. J. Virol. 72, 8463-8471 (1998).
50. Weber, K., Mock, U., Petrowitz, B., Bartsch, U. & Fehse, B. Lentiviral gene ontology (LeGO) vectors equipped with novel drug-selectable fluorescent proteins: new building blocks for cell marking and multi-gene analysis. Gene Ther. 17, 511-520 (2010).
51. Beyer, W. R., Westphal, M., Ostertag, W. & von Laer, D. Oncoretrovirus and lentivirus vectors pseudotyped with lymphocytic choriomeningitis virus glycoprotein: Generation, concentration, and broad host range. J. Virol. 76, 1488-1495 (2002).
52. Weber, K. & Fehse, B. Diva-Fit: A step-by-step manual for generating highresolution graphs and histo-gram overlays of flow cytometry data obtained with FACSDiva software. Cell. Ther. Transplant. 1, 1-4 (2009).
53. Demaison, C. et al. High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. Hum. Gene Ther. 13, 803-813 (2002).