A MicroRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of hemophilia a

Molecular Therapy

Volumn 19, Issue 4, 2011, Pages 723-730

  Matsui, Hideto ^a   Hegadorn, Carol ^a   Ozelo, Margareth ^a   Burnett, Erin ^a   Tuttle, Angie ^a   Labelle, Andrea ^a   McCray Jr , Paul B ^b   Naldini, Luigi ^c   Brown, Brian ^d   Hough, Christine ^a   Lillicrap, David ^a  

^a QUEEN S UNIVERSITY   (Canada)

^b UNIVERSITY OF IOWA   (United States)

^c VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^d MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; GLYCOPROTEIN GP 64; LENTIVIRUS VECTOR; MICRORNA; UNCLASSIFIED DRUG; VIRUS GLYCOPROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BLOOD LEVEL; CONTROLLED STUDY; DISEASE MODEL; GENE EXPRESSION; HEMOPHILIA A; IMMUNE RESPONSE; LENTIVIRINAE; LIVER PARENCHYMA; MACROPHAGE; MOUSE; NONHUMAN; PROMOTER REGION; PSEUDOTYPING; TRANSGENE; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM;

LENTIVIRUS; MURINAE; MUS;

EID: 79953322192     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2010.290     Document Type: Article

References (48)

1. Wight, J and Paisley, S (2003). The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 9: 418-435. (Pubitemid 36874391)
2. Astermark, J, Oldenburg, J, Escobar, M, White, GC 2nd and Berntorp, E (2005). The Malmö International Brother Study (MIBS). Genetic defects and inhibitor development in siblings with severe hemophilia A. Haematologica 90: 924-931.
3. Boekhorst, J, Lari, GR, D'Oiron, R, Costa, JM, Nováková, IR, Ala, FA et al. (2008). Factor VIII genotype and inhibitor development in patients with haemophilia A: highest risk in patients with splice site mutations. Haemophilia 14: 729-735. (Pubitemid 352016028)
4. Astermark, J, Altisent, C, Batorova, A, Diniz, MJ, Gringeri, A, Holme, PA et al. (2010). Non-genetic risk factors and the development of inhibitors in haemophilia: a comprehensive review and consensus report. Haemophilia 16: 747-766.
5. Malhotra, JD, Miao, H, Zhang, K, Wolfson, A, Pennathur, S, Pipe, SW et al. (2008). Antioxidants reduce endoplasmic reticulum stress and improve protein secretion. Proc Natl Acad Sci USA 105: 18525-18530.
6. Dimichele, D (2009). The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapy. Haemophilia 15: 320-328.
7. Ye, P, Thompson, AR, Sarkar, R, Shen, Z, Lillicrap, DP, Kaufman, RJ et al. (2004). Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice. Mol Ther 10: 117-126. (Pubitemid 38878154)
8. Ye, X, Loeb, KR, Stafford, DW, Thompson, AR and Miao, CH (2003). Complete and sustained phenotypic correction of hemophilia B in mice following hepatic gene transfer of a high-expressing human factor IX plasmid. J Thromb Haemost 1: 103-111.
9. Xu, L, Mei, M, Ma, X and Ponder, KP (2007). High expression reduces an antibody response after neonatal gene therapy with B domain-deleted human factor VIII in mice. J Thromb Haemost 5: 1805-1812. (Pubitemid 47288934)
10. Xu, L, Mei, M, Haskins, ME, Nichols, TC, O'donnell, P, Cullen, K et al. (2007). Immune response after neonatal transfer of a human factor IX-expressing retroviral vector in dogs, cats, and mice. Thromb Res 120: 269-280. (Pubitemid 46754415)
11. Park, F, Ohashi, K and Kay, MA (2000). Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver. Blood 96: 1173-1176. (Pubitemid 30616890)
12. Powell, JS, Ragni, MV, White, GC 2nd, Lusher, JM, Hillman-Wiseman, C, Moon, TE et al. (2003). Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood 102: 2038-2045. (Pubitemid 37122377)
13. Roth, DA, Tawa, NE Jr, O'Brien, JM, Treco, DA and Selden, RF (2001). Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med 344: 1735-1742. (Pubitemid 32497027)
14. Vigna, E and Naldini, L (2000). Lentiviral vectors: excellent tools for experimental gene transfer and promising candidates for gene therapy. J Gene Med 2: 308-316.
15. Mátrai, J, Chuah, MK and VandenDriessche, T (2010). Recent advances in lentiviral vector development and applications. Mol Ther 18: 477-490.
16. Levine, BL, Humeau, LM, Boyer, J, MacGregor, RR, Rebello, T, Lu, X et al. (2006). Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci USA 103: 17372-17377. (Pubitemid 44788976)
17. Cartier, N, Hacein-Bey-Abina, S, Bartholomae, CC, Veres, G, Schmidt, M, Kutschera, I et al. (2009). Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326: 818-823.
18. Matsui, H, Shibata, M, Brown, B, Labelle, A, Hegadorn, C, Andrews, C et al. (2009). A murine model for induction of long-term immunologic tolerance to factor VIII does not require persistent detectable levels of plasma factor VIII and involves contributions from Foxp3+ T regulatory cells. Blood 114: 677-685.
19. Matsui, H, Shibata, M, Brown, B, Labelle, A, Hegadorn, C, Andrews, C et al. (2007). Ex vivo gene therapy for hemophilia A that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors. Stem Cells 25: 2660-2669. (Pubitemid 47582759)
20. Van Damme, A, Thorrez, L, Ma, L, Vandenburgh, H, Eyckmans, J, Dell'Accio, F et al. (2006). Efficient lentiviral transduction and improved engraftment of human bone marrow mesenchymal cells. Stem Cells 24: 896-907. (Pubitemid 44464723)
21. Tiede, A, Eder, M, von Depka, M, Battmer, K, Luther, S, Kiem, HP et al. (2003). Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction. Gene Ther 10: 1917-1925. (Pubitemid 37258639)
22. Kootstra, NA, Matsumura, R and Verma, IM (2003). Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther 7(5 Pt 1): 623-631. (Pubitemid 36626242)
23. Follenzi, A, Battaglia, M, Lombardo, A, Annoni, A, Roncarolo, MG and Naldini, L (2004). Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice. Blood 103: 3700-3709. (Pubitemid 38596285)
24. Brown, BD, Cantore, A, Annoni, A, Sergi, LS, Lombardo, A, Della Valle, P et al. (2007). A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood 110: 4144-4152. (Pubitemid 351377775)
25. Chen, CZ, Li, L, Lodish, HF and Bartel, DP (2004). MicroRNAs modulate hematopoietic lineage differentiation. Science 303: 83-86. (Pubitemid 38055775)
26. Brown, BD, Shi, CX, Rawle, FE, Tinlin, S, McKinven, A, Hough, C et al. (2004). Factors influencing therapeutic efficacy and the host immune response to helper-dependent adenoviral gene therapy in hemophilia A mice. J Thromb Haemost 2: 111-118. (Pubitemid 40185627)
27. Vigna, E, Amendola, M, Benedicenti, F, Simmons, AD, Follenzi, A and Naldini, L (2005). Efficient Tet-dependent expression of human factor IX in vivo by a new selfregulating lentiviral vector. Mol Ther 11: 763-775. (Pubitemid 40567981)
28. Bartel, DP (2009). MicroRNAs: target recognition and regulatory functions. Cell 136: 215-233.
29. Brown, BD, Venneri, MA, Zingale, A, Sergi Sergi, L and Naldini, L (2006). Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat Med 12: 585-591.
30. VandenDriessche, T, Thorrez, L, Naldini, L, Follenzi, A, Moons, L, Berneman, Z et al. (2002). Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. Blood 100: 813-822. (Pubitemid 34832606)
31. Pan, D, Gunther, R, Duan, W, Wendell, S, Kaemmerer, W, Kafri, T et al. (2002). Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow. Mol Ther 6: 19-29. (Pubitemid 36124266)
32. Naldini, L, Blömer, U, Gallay, P, Ory, D, Mulligan, R, Gage, FH et al. (1996). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272: 263-267. (Pubitemid 26119138)
33. Brown, BD, Sitia, G, Annoni, A, Hauben, E, Sergi, LS, Zingale, A et al. (2007). In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance. Blood 109: 2797-2805. (Pubitemid 46482074)
34. Kang, Y, Xie, L, Tran, DT, Stein, CS, Hickey, M, Davidson, BL et al. (2005). Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer. Blood 106: 1552-1558. (Pubitemid 41208565)
35. MacKenzie, TC, Kobinger, GP, Kootstra, NA, Radu, A, Sena-Esteves, M, Bouchard, S et al. (2002). Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes. Mol Ther 6: 349-358. (Pubitemid 36305337)
36. Kang, Y, Stein, CS, Heth, JA, Sinn, PL, Penisten, AK, Staber, PD et al. (2002). In vivo gene transfer using a nonprimate lentiviral vector pseudotyped with Ross River Virus glycoproteins. J Virol 76: 9378-9388. (Pubitemid 34919895)
37. Park, F (2003). Correction of bleeding diathesis without liver toxicity using arenaviralpseudotyped HIV-1-based vectors in hemophilia A mice. Hum Gene Ther 14: 1489-1494. (Pubitemid 37268226)
38. Schauber, CA, Tuerk, MJ, Pacheco, CD, Escarpe, PA and Veres, G (2004). Lentiviral vectors pseudotyped with baculovirus gp64 efficiently transduce mouse cells in vivo and show tropism restriction against hematopoietic cell types in vitro. Gene Ther 11: 266-275. (Pubitemid 38220078)
39. Kumar, M, Bradow, BP and Zimmerberg, J (2003). Large-scale production of pseudotyped lentiviral vectors using baculovirus GP64. Hum Gene Ther 14: 67-77. (Pubitemid 36126689)
40. LoDuca, PA, Hoffman, BE and Herzog, RW (2009). Hepatic gene transfer as a means of tolerance induction to transgene products. Curr Gene Ther 9: 104-114.
41. Annoni, A, Brown, BD, Cantore, A, Sergi, LS, Naldini, L and Roncarolo, MG (2009). In vivo delivery of a microRNA-regulated transgene induces antigenspecific regulatory T cells and promotes immunologic tolerance. Blood 114: 5152-5161.
42. Stein, CS, Kang, Y, Sauter, SL, Townsend, K, Staber, P, Derksen, TA et al. (2001). In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors. Mol Ther 3: 850-856. (Pubitemid 32611294)
43. van Til, NP, Markusic, DM, van der Rijt, R, Kunne, C, Hiralall, JK, Vreeling, H et al. (2005). Kupffer cells and not liver sinusoidal endothelial cells prevent lentiviral transduction of hepatocytes. Mol Ther 11: 26-34. (Pubitemid 40011996)
44. Schauber-Plewa, C, Simmons, A, Tuerk, MJ, Pacheco, CD and Veres, G (2005). Complement regulatory proteins are incorporated into lentiviral vectors and protect particles against complement inactivation. Gene Ther 12: 238-245. (Pubitemid 40220625)
45. Montini, E, Cesana, D, Schmidt, M, Sanvito, F, Bartholomae, CC, Ranzani, M et al. (2009). The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J Clin Invest 119: 964-975.
46. Cao, O, Dobrzynski, E, Wang, L, Nayak, S, Mingle, B, Terhorst, C et al. (2007). Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer. Blood 110: 1132-1140. (Pubitemid 47281408)
47. Niwa, H, Yamamura, K and Miyazaki, J (1991). Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 108: 193-199.
48. Bi, L, Lawler, AM, Antonarakis, SE, High, KA, Gearhart, JD and Kazazian, HH Jr (1995). Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet 10: 119-121.