Was cDNA sequences modulate transgene expression of was promoter-driven lentiviral vectors

Human Gene Therapy

Volumn 20, Issue 11, 2009, Pages 1279-1290

  Toscano, Miguel G ^a,b   Benabdellah, Karim ^a,c   Muñoz, Pilar ^d   Frecha, Cecilia ^a,e,f,g   Cobo, Marién ^d   Martín, Francisco ^a,d  

^a INSTITUTO DE PARASITOLOGÍA Y BIOMEDICINA LÓPEZ NEYRA   (Spain)

^b TEMPLE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c CSIC   (Spain)

^d UNIVERSITY OF GRANADA   (Spain)

^e UNIVERSITÉ DE LYON   (France)

^f CENTRE INTERNATIONAL DE RECHERCHE EN INFECTIOLOGIE   (France)

^g UNIVERSITÉ DE LYON   (France)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; ENHANCED GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR; RNA; WISKOTT ALDRICH SYNDROME PROTEIN; GREEN FLUORESCENT PROTEIN; PRIMER DNA; WAS PROTEIN, HUMAN;

ARTICLE; CELL TYPE; CONTROLLED STUDY; ENHANCER REGION; GENE CONTROL; GENE EXPRESSION; GENE INSERTION; GENE SEQUENCE; GENE THERAPY; HEMATOLOGIC DISEASE; HEMATOPOIETIC CELL; HUMAN; HUMAN CELL; NONHUMAN; PROMOTER REGION; RNA PROCESSING; VIRAL GENE DELIVERY SYSTEM; WISKOTT ALDRICH SYNDROME; WISKOTT ALDRICH SYNDROME GENE; WOODCHUCK; FLOW CYTOMETRY; FLUORESCENCE; GENE VECTOR; GENETICS; HEMATOLOGIC DISEASES; HEMATOPOIETIC STEM CELL; JURKAT CELL LINE; LENTIVIRINAE; METABOLISM; PROCEDURES; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; TRANSGENE; WESTERN BLOTTING;

MARMOTA MONAX;

BLOTTING, WESTERN; DNA PRIMERS; DNA, COMPLEMENTARY; FLOW CYTOMETRY; FLUORESCENCE; GENETIC THERAPY; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEMATOLOGIC DISEASES; HEMATOPOIETIC STEM CELLS; HUMANS; JURKAT CELLS; LENTIVIRUS; PROMOTER REGIONS, GENETIC; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; TRANSGENES; WISKOTT-ALDRICH SYNDROME PROTEIN;

EID: 70449115766     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2009.118     Document Type: Article

References (45)

1. Abbasi, A.A., Paparidis, Z., Malik, S., Goode, D.K., Callaway, H., Elgar, G., and Grzeschik, K.H. (2007). Human GLI3 intragenic conserved non-coding sequences are tissue-specific enhancers. PLoS One 2, e366.
2. Barde, I., Zanta-Boussif, M.A., Paisant, S., Leboeuf, M., Rameau, P., Delenda, C., and Danos, O. (2006). Efficient control of gene expression in the hematopoietic system using a single Tet-on inducible lentiviral vector. Mol. Ther. 13, 382-390.
3. Case, S.S., Price, M.A., Jordan, C.T., Yu, X.J., Wang, L., Bauer, G., Haas, D.L., Xu, D., Stripecke, R., Naldini, L., Kohn, D.B., and Crooks, G.M. (1999). Stable transduction of quiescent CD34+ CD38~ human hematopoietic cells by HIV-1-based lentiviral vectors. Proc. Natl. Acad. Sci. U.S.A. 96, 2988-2993.
4. Cui, Y., Narayanan, C.S., Zhou, J., and Kumar, A. (1998). Exon-I is involved in positive as well as negative regulation of human angiotensinogen gene expression. Gene 224, 97-107. (Pubitemid 29023545)
5. Cui, Y., Golob, J., Kelleher, E., YE, Z., Pardoll, D., and Cheng, L. (2002). Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hematopoietic stem/progenitor cells. Blood 99, 399-408.
6. Chang, A.H., and Sadelain, M. (2007). The genetic engineering of hematopoietic stem cells: The rise of lentiviral vectors, the conundrum of the LTR, and the promise of lineage-restricted vectors. Mol. Ther. 15, 445-456.
7. Demaison, C, Parsley, K., Brouns, G., Scherr, M., Battmer, K., Kinnon, C, Grez, M., and Thrasher, A.J. (2002). High-level transduction and gene expression in hematopoietic repop-ulating cells using a human immunodeficiency [correction of imunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. Hum. Gene Ther. 13, 803-813.
8. Donello, J.E., Loeb, J.E., and Hope, T.J. (1998). Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element. J. Virol. 72, 5085-5092.
9. Frecha, C, Toscano, M.G., Costa, C, Saez-Lara, M.J., Cosset, F.L., Verhoeyen, E., and Martin, F. (2008). Improved lentiviral vectors for Wiskott-Aldrich syndrome gene therapy mimic endogenous expression profiles throughout haematopoiesis. Gene Ther. 15, 930-941.
10. Gallego, M.D., Santamaria, M., Pena, J., and Molina, I.J. (1997). Defective actin reorganization and polymerization of Wiskott-Aldrich T cells in response to CD3-mediated stimulation. Blood 90, 3089-3097.
11. Garcia-Pacheco, J.M., Oliver, C., Kimatrai, M., Blanco, F.J., and Olivares, E.G. (2001). Human decidual stromal cells express CD34 and STRO-1 and are related to bone marrow stromal precursors. Mol. Hum. Reprod. 7, 1151-1157.
12. Gaszner, M., and Felsenfeld, G. (2006). Insulators: Exploiting transcriptional and epigenetic mechanisms. Nat. Rev. 7, 703-713.
13. Goverdhana, S., Puntel, M., Xiong, W., ZIRGER, J.M., Barcia, C., Curtin, J.F., Soffer, E.B., Mondkar, S., King, G.D., Hu, J., Sciascia, S.A., Candolfi, M., Greengold, D.S., Lowenstein, P.R., and Castro, M.G. (2005). Regulatable gene expression systems for gene therapy applications: Progress and future challenges. Mol. Ther. 12, 189-211.
14. Han, X.D., Lin, C., Chang, J., Sadelain, M., and Kan, Y.W. (2007). Fetal gene therapy of a-thalassemia in a mouse model. Proc. Natl. Acad. Sci. U.S.A. 104, 9007-9011.
15. Hawley, R.G. (2001). Progress toward vector design for he-matopoietic stem cell gene therapy. Curr, Gene Ther. 1, 1-17.
16. Hlavaty, J., Schittmayer, M., Stracke, A., Jandl, G., Knapp, E., Felber, B.K., Salmons, B., Gunzburg, W.H., and Renner, M. (2005). Effect of posttranscriptional regulatory elements on transgene expression and virus production in the context of retrovirus vectors. Virology 341, 1-11.
17. Jager, U., Zhao, Y., and Porter, C.D. (1999). Endothelial cell-specific transcriptional targeting from a hybrid long terminal repeat retrovirus vector containing human prepro-endothelin-1 promoter sequences. J. Virol. 73, 9702-9709.
18. Kobayashi, M., Nishikawa, K., and Yamamoto, M. (2001). Hematopoietic regulatory domain of gata1 gene is positively regulated by GATA1 protein in zebrafish embryos. Development 128, 2341-2350.
19. Levine, M., and Tjian, R. (2003). Transcription regulation and animal diversity. Nature 424, 147-151.
20. Lin, C.J., and Tam, R.C. (2001). Transcriptional regulation of CD28 expression by CD28GR, a novel promoter element located in exon 1 of the CD28 gene. J. Immunol 166, 6134-6143.
21. Lotti, F., Menguzzato, E., Rossi, C., Naldini, L., Ailles, L., Ma-vilio, F., and Ferrari, G. (2002). Transcriptional targeting of lentiviral vectors by long terminal repeat enhancer replacement. J. Virol. 76, 3996-4007.
22. Lutzko, C., Senadheera, D., Skelton, D., Petersen, D., and Kohn, D.B. (2003). Lentivirus vectors incorporating the immuno-globulin heavy chain enhancer and matrix attachment regions provide position-independent expression in B lymphocytes. J. Virol. 77, 7341-7351.
23. Marodon, G., Mouly, E., Blair, E.J., Frisen, C., Lemoine, F.M., and Klatzmann, D. (2003). Specific transgene expression in human and mouse CD4+ cells using lentiviral vectors with regulatory sequences from the CD4 gene. Blood 101, 3416-3423.
24. Martin, F., Toscano, M.G., Blundell, M., Frecha, C., Srivastava, G.K., Santamaria, M., Thrasher, A.J., and Molina, I.J. (2005). Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences. Gene Ther. 12, 715-723.
25. Mastroyiannopoulos, N.P., Feldman, M.L., Uney, J.B., Mahade-van, M.S., and Phylactou, L.A. (2005). Woodchuck post-transcriptional element induces nuclear export of myotonic dystrophy 30 untranslated region transcripts. EMBO Rep 6, 458-463.
26. Modlich, U., Bohne, J., Schmidt, M., Von Kalle, C., Knoss, S., Schambach, A., and Baum, C. (2006). Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood 108, 2545-2553.
27. Moreau, T., Bardin, F., Imbert, J., Chabannon, C., and Tonnelle, C. (2004). Restriction of transgene expression to the B-lymphoid progeny of human lentivirally transduced CD34+ cells. Mol. Ther. 10, 45-56.
28. Moreau-Gaudry, F., Xia, P., Jiang, G., Perelman, N.P., Bauer, G., Ellis, J., Surinya, K.H., Mavilio, F., Shen, C.K., and Malik, P. (2001). High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors. Blood 98, 2664-2672.
29. Neil, S., Martin, F., Ikeda, Y., and Collins, M. (2001). Postentry restriction to human immunodeficiency virus-based vector transduction in human monocytes. J. Virol. 75, 5448-5456.
30. Oertel, M., Rosencrantz, R., Chen, Y.Q., Thota, P.N., Sandhu, J.S., Dabeva, M.D., Pacchia, A.L., Adelson, M.E., Dougherty, J.P., and Shafritz, D.A. (2003). Repopulation of rat liver by fetal hepatoblasts and adult hepatocytes transduced ex vivo with lentiviral vectors. Hepatology 37, 994-1005.
31. Osborne, C.S., Chakalova, L., Brown, K.E., Carter, D., Horton, A., Debrand, E., Goyenechea, B., Mitchell, J.A., Lopes, S., Reik, W., and Fraser, P. (2004). Active genes dynamically colocalize to shared sites of ongoing transcription. Nat. Genet. 36, 1065-1071.
32. Robert-Richard, E., Richard, E., Malik, P., Ged, C., De Verneuil, H., and Moreau-Gaudry, F. (2007). Murine retroviral but not human cellular promoters induce in vivo erythroid-specific deregulation that can be partially prevented by insulators. Mol. Ther. 15, 173-182.
33. Sakonju, S., Bogenhagen, D.F., and Brown, D.D. (1980). A control region in the center of the 5S RNA gene directs specific initiation of transcription. I. The 50 border of the region. Cell 19, 13-25. (Pubitemid 10154209)
34. Saur, D., Seidler, B., Paehge, H., Schusdziarra, V., and Allescher, H.D. (2002). Complex regulation of human neuronal nitric-oxide synthase exon 1c gene transcription: Essential role of Sp and ZNF family members of transcription factors. J. Biol. Chem. 277, 25798-25814.
35. Seshasayee, D., Geiger, J.N., Gaines, P., and Wojchowski, D.M. (2000). Intron 1 elements promote erythroid-specific GATA-1 gene expression. J. Biol. Chem. 275, 22969-22977.
36. Sternberg, E.A., Spizz, G., Perry, W.M., Vizard, D., Weil, T., and Olson, E.N. (1988). Identification of upstream and intragenic regulatory elements that confer cell-type-restricted and differentiation-specific expression on the muscle creatine ki-nase gene. Mol. Cell. Biol. 8, 2896-2909.
37. Szulc, J., Wiznerowicz, M., Sauvain, M.O., Trono, D., and Aebischer, P. (2006). A versatile tool for conditional gene expression and knockdown. Nat. Methods 3, 109-116.
38. Toscano, M.G., Frecha, C., Benabdellah, K., Cobo, M., Blundell, M., Thrasher, A.J., Garcia-Olivares, E., Molina, I.J., and Martin, F. (2008). Hematopoietic-specific lentiviral vectors circumvent cellular toxicity due to ectopic expression of Wiskott-Aldrich syndrome protein. Hum. Gene Ther. 19, 179-197.
39. Werner, M., Kraunus, J., Baum, C., and Brocker, T. (2004). B-cell-specific transgene expression using a self-inactivating retroviral vector with human CD19 promoter and viral post-transcriptional regulatory element. Gene Ther. 11, 992-1000.
40. Wiznerowicz, M., and Trono, D. (2005). Harnessing HIV for therapy, basic research and biotechnology. Trends Biotechnol. 23, 42-47.
41. Wong, E.Y., Lin, J., Forget, B.G., Bodine, D.M., and Gallagher, P.G. (2004). Sequences downstream of the erythroid promoter are required for high level expression of the human a-spectrin gene. J. Biol. Chem. 279, 55024-55033.
42. Xu, L., Wallen, R., Patel, V., and Depinho, R.A. (1993). Role of first exon=intron sequences in the regulation of myc family oncogenic potency. Oncogene 8, 2547-2553. (Pubitemid 23252385)
43. Zufferey, R., Dull, T., Mandel, R.J., Bukovsky, A., Quiroz, D., Naldini, L., and Trono, D. (1998). Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 72, 9873-9880.
44. Zufferey, R., Donello, J.E., Trono, D., and Hope, T.J. (1999). Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by ret-roviral vectors. J. Virol. 73, 2886-2892.
45. Zychlinski, D., Schambach, A., Modlich, U., Maetzig, T., Meyer, J., Grassman, E., Mishra, A., and Baum, C. (2008). Physiological promoters reduce the genotoxic risk of integrating gene vectors. Mol. Ther. 16, 718-725