Lentiviral vectors encoding zinc-finger nucleases specific for the model target locus HPRT1

Methods in Molecular Biology

Volumn 1114, Issue , 2014, Pages 181-199

  Pelascini, Laetitia P L ^a   Gonçalves, Manuel A F V ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

DNA repair; Double stranded DNA breaks; Gene correction; Gene knockout; Gene targeting; HPRT1; Lentiviral vectors; Zinc finger nucleases

Indexed keywords

HYPOXANTHINE PHOSPHORIBOSYLTRANSFERASE; LENTIVIRUS VECTOR; RESTRICTION ENDONUCLEASE; ZINC FINGER NUCLEASE; HYPOXANTHINE PHOSPHORIBOSYLTRANSFERASE 1; UNCLASSIFIED DRUG;

ARTICLE; DNA TRANSFECTION; GENE DISRUPTION; GENE LOCUS; GENE TARGETING; GENOTYPE; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; PROTEIN PURIFICATION; TITRIMETRY; ULTRACENTRIFUGATION; VIRAL GENE DELIVERY SYSTEM; CARCINOMA CELL; CONTROLLED STUDY; DNA CLEAVAGE; DOUBLE STRANDED DNA BREAK; GENETIC VARIABILITY; HUMAN; HUMAN CELL; VIRUS TITRATION;

MAMMALIA;

ANIMALS; DNA BREAKS, DOUBLE-STRANDED; DNA REPAIR; ENDONUCLEASES; GENE TARGETING; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GENOTYPING TECHNIQUES; HUMANS; HYPOXANTHINE PHOSPHORIBOSYLTRANSFERASE; LENTIVIRUS; TRANSDUCTION, GENETIC; ZINC FINGERS;

EID: 84898945717     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-62703-761-7_12     Document Type: Article

References (30)

1. Klug A (2010) The discovery of zinc fi ngers and their applications in gene regulation and genome manipulation. Annu Rev Biochem 79:213-231
2. Silva G, Poirot L, Galetto R, Smith J, Montoya G, Duchateau P, Pâques F (2011) Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy. Curr Gene Ther 11:11-27
3. Mussolino C, Cathomen T (2012) TALE nucleases: tailored genome engineering made easy. Curr Opin Biotechnol 23:644-650
4. Perez-Pinera P, Ousterout DG, Gersbach CA (2012) Advances in targeted genome editing. Curr Opin Chem Biol 16:268-277
5. Brunet E, Simsek D, Tomishima M, DeKelver R, Choi VM, Gregory P, Urnov F, Weinstock DM, Jasin M (2009) Chromosomal translocations induced at specifi ed loci in human stem cells. Proc Natl Acad Sci USA 106:10620-10625
6. Naldini L (2011) Ex vivo gene transfer and correction for cell-based therapies. Nat Rev Genet 12:301-315
7. Kim YG, Cha J, Chandrasegaran S (1996) Hybrid restriction enzymes: zinc fi nger fusions to Fok I cleavage domain. Proc Natl Acad Sci USA 93:1156-1160
8. Perez EE, Wang J, Miller JC, Jouvenot Y, Kim KA, Liu O, Wang N, Lee G, Bartsevich VV, Lee YL, Guschin DY, Rupniewski I, Waite AJ, Carpenito C, Carroll RG, Orange JS, Urnov FD, Rebar EJ, Ando D, Gregory PD, Riley JL, Holmes MC, June CH (2008) Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-fi nger nucleases. Nat Biotechnol 26:808-816
9. Dropuli? B (2011) Lentiviral vectors: their molecular design, safety, and use in laboratory and preclinical research. Hum Gene Ther 22:649-657
10. Cronin J, Zhang XY, Reiser J (2005) Altering the tropism of lentiviral vectors through pseudotyping. Curr Gene Ther 5:387-398, Erratum in: Curr Gene Ther 5:531, 2005
11. Philpott NJ, Thrasher AJ (2007) Use of nonintegrating lentiviral vectors for gene therapy. Hum Gene Ther 18:483-489
12. Boussif O, Lezoualc'h F, Zanta MA, Mergny MD, Scherman D, Demeneix B, Behr JP (1995) A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci USA 92:7297-7301
13. Kuroda H, Kutner RH, Bazan NG, Reiser J (2009) Simplifi ed lentivirus vector production in protein-free media using polyethyleniminemediated transfection. J Virol Methods 157:113-12
14. Segura MM, Garnier A, Durocher Y, Ansorge S, Kamen A (2010) New protocol for lentiviral vector mass production. Methods Mol Biol 614:39-52
15. Askar SF, Bingen BO, Swildens J, Ypey DL, van der Laarse A, Atsma DE, Zeppenfeld K, Schalij MJ, de Vries AAF, Pijnappels DA (2012) Connexin43 silencing in myofi broblasts prevents arrhythmias in myocardial cultures: role of maximal diastolic potential. Cardiovasc Res 93:434-444
16. Pelascini LP, Janssen JM, Gonçalves MAFV (2013) Histone deacetylase inhibition activates transgene expression from integration-defective lentiviral vectors in dividing and non-dividing cells. Hum Gene Ther 24:78-96
17. Torres RJ, Puig JG (2007) Hypoxanthineguanine phosophoribosyltransferase (HPRT) defi ciency: Lesch-Nyhan syndrome. Orphanet J Rare Dis 2:e48
18. Szybalski W (1992) Use of the HPRT gene and the HAT selection technique in DNA-mediated transformation of mammalian cells: First steps toward developing hybridoma techniques and gene therapy. Bioessays 14:495-500
19. Milstein C (1999) The hybridoma revolution: an offshoot of basic research. Bioessays 21:966-973
20. Doetschman T, Gregg RG, Maeda N, Hooper ML, Melton DW, Thompson S, Smithies O (1987) Targetted correction of a mutant HPRT gene in mouse embryonic stem cells. Nature 330:576-578
21. Thomas KR, Capecchi MR (1987) Site-directed mutagenesis by gene targeting in mouse embryo-derived stem cells. Cell 51:503-512
22. Miller AD, Jolly DJ, Friedmann T, Verma IM (1983) A transmissible retrovirus expressing human hypoxanthine phosphoribosyltransferase (HPRT): gene transfer into cells obtained from humans defi cient in HPRT. Proc Natl Acad Sci USA 80:4709-4713
23. Gonçalves MAFV (2005) A concise peer into the background, initial thoughts and practices of human gene therapy. Bioessays 17:506-517
24. Charrier S, Stockholm D, Seye K, Opolon P, Taveau M, Gross DA, Bucher-Laurent S, Delenda C, Vainchenker W, Danos O, Galy A (2005) A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice. Gene Ther 12:597-606
25. Miller JC, Holmes MC, Wang J, Guschin DY, Lee YL, Rupniewski I, Beausejour CM, Waite AJ, Wang NS, Kim KA, Gregory PD, Pabo CO, Rebar EJ (2007) An improved zinc-fi nger nuclease architecture for highly specifi c genome editing. Nat Biotechnol 25:778-785
26. Higashikawa F, Chang L (2001) Kinetic analyses of stability of simple and complex retroviral vectors. Virology 280:124-131
27. Wanisch K, Yáñez-Muñoz RJ (2009) Integration-defi cient lentiviral vectors: a slow coming of age. Mol Ther 17:1316-1332
28. Giry-Laterrière M, Verhoeyen E, Salmon P (2011) Lentiviral vectors. Methods Mol Biol 737:183-209
29. Hou Y, Zhang H, Miranda L, Lin S (2010) Serious overestimation in quantitative PCR by circular (supercoiled) plasmid standard: microalgal pcna as the model gene. PLoS One 5(3):e9545
30. Delenda C, Gaillard C (2005) Real-time quantitative PCR for the design of lentiviral vector analytical assays. Gene Ther 12:S36-S50