Optimizing delivery and expression of designer nucleases for genome engineering

Human Gene Therapy Methods

Volumn 24, Issue 6, 2013, Pages 329-332

  Chuah, Marinee K ^a,b   Vandendriessche, Thierry ^a,b  

^a VRIJE UNIVERSITEIT BRUSSEL   (Belgium)

^b UNIVERSITY OF LEUVEN   (Belgium)

Author keywords

[No Author keywords available]

Indexed keywords

DOUBLE STRANDED DNA; HISTONE DEACETYLASE INHIBITOR; INTEGRATION DEFECTIVE LENTIVIRAL VECTOR; LENTIVIRUS VECTOR; TRICHOSTATIN A; UNCLASSIFIED DRUG; VORINOSTAT; ZINC FINGER NUCLEASE;

CHROMATIN ASSEMBLY AND DISASSEMBLY; CHROMATIN CONDENSATION; DNA CLEAVAGE; DNA END JOINING REPAIR; DNA REPAIR; EPIGENETIC REPRESSION; GENE TARGETING; GENETIC ENGINEERING; HELA CELL; HUMAN; MYOBLAST; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PRIORITY JOURNAL; REVIEW; STABLE EXPRESSION; TRANSGENE; TRANSIENT EXPRESSION;

CHROMATIN ASSEMBLY AND DISASSEMBLY; DEOXYRIBONUCLEASES; GENETIC ENGINEERING; GENETIC VECTORS; GENOME, HUMAN; HISTONE DEACETYLASE INHIBITORS; HUMANS; LENTIVIRUS; ZINC FINGERS;

EID: 84890367152     PISSN: 19466536     EISSN: 19466544     Source Type: Journal    
DOI: 10.1089/hgtb.2013.166     Document Type: Review

References (35)

1. Anguela, X.M., Sharma, R., Doyon, Y., et al. (2013). Robust ZFNmediated genome editing in adult hemophilic mice. Blood 122, 3283-3287
2. Carbery, I.D., Ji, D., Harrington, A., et al. (2010). Targeted genome modification in mice using zinc-finger nucleases. Genetics 186, 451-459
3. Coluccio, A., Miselli, F., Lombardo, A., et al. (2013). Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination. Mol. Ther. 21, 1695-1704
4. Cradick, T.J., Fine, E.J., Antico, C.J., and Bao, G. (2013). CRISPR/ Cas9 systems targeting b-globin and CCR5 genes have substantial off-Target activity. Nucleic Acids Res. 41, 9584-9592
5. Daboussi, F., Zaslavskiy, M., Poirot, L., et al. (2012). Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases. Nucleic Acids Res 40, 6367-6379
6. Di Micco, S., Chini, M.G., Terracciano, S., et al. (2013). Structural basis for the design and synthesis of selective HDAC inhibitors. Bioorg. Med. Chem. 21, 3795-3807
7. Fu, Y., Foden, J.A., Khayter, C., et al. (2013). High-frequency offtarget mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat. Biotechnol. 31, 822-826
8. Gaj, T., Gersbach, C.A., and Barbas, C.F., 3rd. (2013). ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol. 31, 397-405
9. Grau, J., Boch, J., and Posch, S. (2013). TALENoffer: Genomewide TALEN off-Target prediction. Bioinformatics 29, 2931-2932
10. Händel, E.M., Gellhaus, K., Khan, K., et al. (2012). Versatile and efficient genome editing in human cells by combining zincfinger nucleases with adeno-Associated viral vectors. Hum. Gene Ther. 23, 321-329
11. Hofer, U., Henley, J.E., Exline, C.M., et al. (2013). Pre-clinical modeling of CCR5 knockout in human hematopoietic stem cells by zinc finger nucleases using humanized mice. J. Infect. Dis. 208 Suppl 2, S160-S164
12. Holkers,M.,Maggio, I., Liu, J., et al. (2013). Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells. Nucleic Acids Res. 41, e63
13. Holt, N., Wang, J., Kim, K., 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
14. Izmiryan, A., Basmaciogullari, S., Henry, A., et al. (2011). Efficient gene targeting mediated by a lentiviral vector-Associated meganuclease. Nucleic Acids Res. 39, 7610-7619
15. Li, H., Haurigot, V., Doyon, Y., et al. (2011). In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature 475, 217-221
16. 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, 1259-1269
17. Lombardo, A., Genovese, P., Beausejour, C.M., et al. (2007). Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat. Biotechnol. 25, 1298-1306
18. Lombardo, A., Cesana, D., Genovese, P., et al. (2011). Sitespecific integration and tailoring of cassette design for sustainable gene transfer. Nat. Methods 8, 861-869
19. Maier, D.A., Brennan, A.L., 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, 245-258
20. Mali, P., Yang, L., Esvelt, K.M., et al. (2013). RNA-guided human genome engineering via Cas9. Science 339, 823-826
21. Mátés, L., Chuah, M.K., Belay, E., et al. (2009). Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates. Nat. Genet. 41, 753-761
22. Má trai, J., Chuah, M.K., and VandenDriessche, T. (2010). Recent advances in lentiviral vector development and applications. Mol. Ther. 18, 477-490
23. Má trai, J., Cantore, A., Bartholomae, C.C., et al. (2011). Hepatocyte-Targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk. Hepatology 53, 1696-1707
24. Moldt, B., Staunstrup, N.H., Jakobsen, M., et al. (2008). Genomic insertion of lentiviral DNA circles directed by the yeast Flp recombinase. BMC Biotechnol. 8, e60
25. Moldt, B., Miskey, C., Staunstrup, N.H., et al. (2011). Comparative genomic integration profiling of Sleeping Beauty transposons mobilized with high efficacy from integrase-defective lentiviral vectors in primary human cells. Mol. Ther. 19, 1499-1510
26. Pelascini, L.P., Janssen, J.M., and Gonçalves, M.A.F.V. (2013a). Histone deacetylase inhibition activates transgene expression from integration-defective lentiviral vectors in dividing and non-dividing cells. Hum. Gene Ther. 24, 78-96
27. Pelascini, L., Maggio, I., Liu, J., et al. (2013b) Histone deacetylase inhibition rescues gene knockout levels achieved with integrase-defective lentiviral vectors encoding zinc-finger nucleases. Hum. Gene Ther. [Epub ahead of print]
28. Perez, E.E., Wang, J., Miller, J.C., et al. (2008). Establishment of HIV-1 resistance in CD4 + T cells by genome editing using zinc-finger nucleases. Nat. Biotechnol. 26, 808-816
29. Provasi, E., Genovese, P., Lombardo, A., et al. (2012). Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer. Nat. Med. 18, 807-815
30. Sander, J.D., Ramirez, C.L., Linder, S.J., et al. (2013). In silico abstraction of zinc finger nuclease cleavage profiles reveals an expanded landscape of off-Target sites. Nucleic Acids Res. 41, e181
31. Staunstrup, N.H., Moldt, B., Mátés, L., et al. (2009). Hybrid lentivirus-Transposon vectors with a random integration profile in human cells. Mol. Ther. 17, 1205-1214
32. Torikai, H., Reik, A., Liu, P.Q., et al. (2012). A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-Antigen-receptor and eliminate expression of endogenous TCR. Blood 119, 5697-5705
33. van Rensburg, R., Beyer, I., Yao, X.Y., et al. (2013). Chromatin structure of two genomic sites for targeted transgene integration in induced pluripotent stem cells and hematopoietic stem cells. Gene Ther. 20, 201-214
34. Vink, C.A., Gaspar, H.B., Gabriel, R., et al. (2009). Sleeping beauty transposition from nonintegrating lentivirus. Mol. Ther. 17, 1197-1204
35. Yáñez-Muñoz, R.J., Balaggan, K.S., MacNeil, A., et al (2006). Effective gene therapy with nonintegrating lentiviral vectors. Nat. Med.e12, 348-353