An integration-defective lentivirus-based resource for site-specific targeting of an edited safe-harbour locus in the human genome

Gene Therapy

Volumn 21, Issue 4, 2014, Pages 343-352

  Torres, R ^a   Garcia, A ^a   Jimenez, M ^a   Rodriguez, S ^b   Ramirez, J C ^a  

^a CENTRO NACIONAL DE INVESTIGACIONES CARDIOVASCULARES CNIC   (Spain)

^b Centro Nacional de Investigaciones Oncológicas   (Spain)

Author keywords

non integrative lentivirus; RMCE; site specific integration

Indexed keywords

CRE RECOMBINASE; INTEGRASE DEFECTIVE LENTIVIRUS; LENTIVIRUS VECTOR; RECOMBINASE; UNCLASSIFIED DRUG; ZINC FINGER NUCLEASE; INTEGRASE;

ARTICLE; GENE CASSETTE; GENE LOCUS; GENETIC ANALYSIS; HUMAN; HUMAN CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; TRANSGENICS; ANIMAL; CELL DIFFERENTIATION; GENE TARGETING; GENE THERAPY; GENETICS; HUMAN GENOME; LENTIVIRINAE; MOUSE; PLURIPOTENT STEM CELL;

LENTIVIRUS; MUS;

ANIMALS; CELL DIFFERENTIATION; GENE TARGETING; GENETIC THERAPY; GENOME, HUMAN; HUMANS; INDUCED PLURIPOTENT STEM CELLS; INTEGRASES; LENTIVIRUS; MICE;

EID: 84897570809     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2014.1     Document Type: Article

References (25)

1. Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 1995; 270: 475-480.
2. Hockemeyer D, Soldner F, Beard C, Gao Q, Mitalipova M, DeKelver RC et al. Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 2009; 27: 851-857.
3. Zou J, Maeder ML, Mali P, Pruett-Miller SM, Thibodeau-Beganny S, Chou B-K et al. Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells. Cell Stem Cell 2009; 5: 97-110.
4. Chapdelaine P, Pichavant C, Rousseau J, P Acirc Ques F, Tremblay JP. Meganucleases can restore the reading frame of a mutated dystrophin. Gene Ther 2010; 17: 846-858.
5. Miller JC, Tan S, Qiao G, Barlow KA, Wang J, Xia DF et al. A TALE nuclease architecture for efficient genome editing. Nat Biotechnol 2011; 29: 143-148.
6. Mali P, Yang L, Esvelt KM, Aach J, Guell M, DiCarlo JE et al. RNA-guided human genome engineering via Cas9. Science 2013; 339: 823-826.
7. Turan S, Galla M, Ernst E, Qiao J, Voelkel C, Schiedlmeier B et al. Recombinase-mediated cassette exchange (RMCE): traditional concepts and current challenges. J Mol Biol 2011; 407: 193-221.
8. DeKelver RC, Choi VM, Moehle EA, Paschon DE, Hockemeyer D, Meijsing SH et al. Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome. Genome Res 2010; 20: 1133-1142.
9. Sadelain M, Papapetrou EP, Bushman FD. Safe harbours for the integration of new DNA in the human genome. Nat Rev Cancer 2012; 12: 51-58.
10. Torres R, Garćia A, Payá M, Ramirez JC. Non-integrative lentivirus drives high-frequency cre-mediated cassette exchange in human cells. PLoS One 2011; 6: e19794.
11. Lombardo A, Genovese P, Beausejour CM, Colleoni S, Lee Y-L, Kim KA et al. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol 2007; 25: 1298-1306.
12. Kolb AF. Selection-marker-free modification of the murine beta-casein gene using a lox2272 [correction of lox2722] site. Anal Biochem 2001; 290: 260-271.
13. Qiao J, Oumard A, Wegloehner W, Bode J. Novel tag-and-exchange (RMCE) strategies generate master cell clones with predictable and stable transgene expression properties. J Mol Biol 2009; 390: 579-594.
14. Silver DP, Livingston DM. Self-excising retroviral vectors encoding the Cre recombinase overcome Cre-mediated cellular toxicity. Mol Cell 2001; 8: 233-243.
15. Fehse B, Kustikova OS, Bubenheim M, Baum C. Pois(s)on-it's a question of dose. Gene Ther 2004; 11: 879-881.
16. Muzumdar MD, Tasic B, Miyamichi K, Li L, Luo L. A global double-fluorescent Cre reporter mouse. genesis 2007; 45: 593-605.
17. Smith JR, Maguire S, Davis LA, Alexander M, Yang F, Chandran S et al. Robust, persistent transgene expression in human embryonic stem cells is achieved with AAVS1-targeted integration. Stem Cells 2008; 26: 496-504.
18. van Rensburg R, Beyer I, Yao XY, Wang H, Denisenko O, Li Z-Y et al. Chromatin structure of two genomic sites for targeted transgene integration in induced pluripotent stem cells and hematopoietic stem cells. Gene Ther 2013; 20: 201-214.
19. Wanisch K, Yáñez-Muñoz RJ. Integration-deficient lentiviral vectors: a slow coming of age. Mol Ther 2009; 17: 1316-1332.
20. Brown JP, Wei W, Sedivy JM. Bypass of senescence after disruption of p21CIP1/WAF1 gene in normal diploid human fibroblasts. Science 1997; 277: 831-834.
21. Marión RM, Strati K, Li H, Murga M, Blanco R, Ortega S et al. A p53-mediated DNA damage response limits reprogramming to ensure iPS cell genomic integrity. Nature 2009; 460: 1149-1153.
22. Li H, Collado M, Villasante A, Strati K, Ortega S, Cañamero M et al. The Ink4/Arf locus is a barrier for iPS cell reprogramming. Nature 2009; 460: 1136-1139.
23. Hockemeyer D, Jaenisch R. Gene targeting in human pluripotent cells. Cold Spring Harbor Symposia on Quantitative Biology 2011; 75: 201-209.
24. González F, Boué S, Belmonte J. Methods for making induced pluripotent stem cells: reprogramming a la carte. Nat Rev Genet 2011; 12: 231-242.
25. Neff NF, Quake SR, Lu R, Weissman IL. Tracking single hematopoietic stem cells in vivo using high-throughput sequencing in conjunction with viral genetic barcoding. Nat Biotechnol 2011; 29: 928-933.