Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes

Molecular Therapy

Volumn 6, Issue 3, 2002, Pages 349-358

  MacKenzie, Tippi C ^a   Kobinger, Gary P ^b   Kootstra, Neeltje A ^c   Radu, Antoneta ^a   Sena Esteves, Miguel ^a   Bouchard, Sarah ^a   Wilson, James M ^b   Verma, Inder M ^c   Flake, Alan W ^a  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b Institute for Human Gene Therapy   (United States)

^c SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

Author keywords

CMV promoter; Ebola; Fetal therapy; Lentivirus; Liver transduction; Mokola; Muscle transduction; Pseudotyping; VSV G

Indexed keywords

BETA GALACTOSIDASE; GLYCOPROTEIN; LENTIVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; CYTOMEGALOVIRUS; EBOLA VIRUS; FETUS; GENE TRANSFER; GENETIC TRANSDUCTION; HEART MUSCLE CELL; HISTOLOGY; INJECTION SITE; INTERMETHOD COMPARISON; LIVER CELL; MOUSE; MUSCLE CELL; MUSCULAR DYSTROPHY; NONHUMAN; TISSUE DISTRIBUTION; VESICULAR STOMATITIS VIRUS; VIRAL GENE DELIVERY SYSTEM;

ANIMALIA; CUCUMBER MOSAIC VIRUS; CYTOMEGALOVIRUS; EBOLA VIRUS; HUMAN HERPESVIRUS 5; LENTIVIRUS; MOKOLA VIRUS; VESICULAR STOMATITIS VIRUS;

EID: 0346035738     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2002.0681     Document Type: Article

References (39)

1. Yang, E. Y., Flake, A. W., and Adzick, N. S. (1999). Prospects for fetal gene therapy. Semin. Perinatol. 23: 524-534.
2. Herzog, R. W., and High, K. A. (1998). Problems and prospects in gene therapy for hemophilia. Curr. Opin. Hematol. 5: 321-326.
3. Yang, E. Y., et al. (1999). Persistent postnatal transgene expression in both muscle and liver after fetal injection of recombinant adenovirus. J. Pediatr. Surg. 34: 766-772.
4. Yang, E. Y., Cass, D. L., Sylvester, K. G., Wilson, J. M., and Adzick, N. S. (1999). Fetal gene therapy: efficacy, toxicity, and immunologic effects of early gestation recombinant adenovirus. J. Pediatr. Surg. 34: 235-241.
5. Tran, N. D., et al. (2000). In utero transfer and expression of exogenous genes in sheep. Exp. Hematol. 28: 17-30.
6. Tran, N. D., et al. (2001). Induction of stable prenatal tolerance to β-galactosidase by in utero gene transfer into preimmune sheep fetuses. Blood 97: 3417-3423.
7. Porada, C. D., et al. (1998). In utero gene therapy: transfer and long-term expression of the bacterial neo(r) gene in sheep after direct injection of retroviral vectors into preimmune fetuses. Hum. Gene Ther. 9: 1571-1585.
8. Porada, C. D., Tran, N. D., Zhao, Y., Anderson, W. F., and Zanjani, E. D. (2000). Neonatal gene therapy. Transfer and expression of exogenous genes in neonatal sheep following direct injection of retroviral vectors into the bone marrow space. Exp. Hematol. 28: 642-650.
9. Themis, M., et al. (1999). Successful expression of β-galactosidase and factor IX transgenes in fetal and neonatal sheep after ultrasound-guided percutaneous adenovirus vector administration into the umbilical vein. Gene Ther. 6: 1239-1248.
10. Mitchell, M., Jerebtsova, M., Batshaw, M. L., Newman, K., and Ye, X. (2000). Long-term gene transfer to mouse fetuses with recombinant adenovirus and adeno-associated virus (AAV) vectors. Gene Ther. 7: 1986-1992.
11. Sekhon, H. S., and Larson, J. E. (1995). In utero gene transfer into the pulmonary epithelium. Nat. Med. 1: 1201-1203.
12. Larson, J. E., Morrow, S. L., Happel, L., Sharp, J. F., and Cohen, J. C. (1997). Reversal of cystic fibrosis phenotype in mice by gene therapy in utero. Lancet 349: 619-620.
13. Lipshutz, G. S., Flebbe-Rehwaldt, L., and Gaensler, K. M. (2000). Reexpression following readministration of an adenoviral vector in adult mice after initial in utero adenoviral administration. Mol. Ther. 2: 374-380, doi.10.1006/mthe.2000.0133.
14. Schneider, H., et al. (2002). Sustained delivery of therapeutic concentrations of human clotting factor IX - a comparison of adenoviral and AAV vectors administered in utero. J. Gene. Med. 4: 46-53.
15. Tarantal, A. F., et al. (2001). Rhesus monkey model for fetal gene transfer: studies with retroviral-based vector systems. Mol. Ther. 3: 128-138, doi.10.1006/mthe.2000.0255.
16. Lipshutz, G. S., et al. (2001). In utero delivery of adeno-associated viral vectors: intraperitoneal gene transfer produces long-term expression. Mol. Ther. 3: 284-292, doi.10.1006/mthe.2001.0267.
17. Loser, P., Jennings, G. S., Strauss, M., and Sandig, V. (1998). Reactivation of the previously silenced cytomegalovirus major immediate-early promoter in the mouse liver: involvement of NFκB. J. Virol. 72: 180-190.
18. Clapp, D. W., Dumenco, L. L., Hatzoglou, M., and Gerson, S. L. (1991). Fetal liver hematopoietic stem cells as a target for in utero retroviral gene transfer. Blood 78: 1132-1139.
19. Clapp, D. W., Freie, B., Lee, W. H., and Zhang, Y. Y. (1995). Molecular evidence that in situ-transduced fetal liver hematopoietic stem/progenitor cells give rise to medullary hematopoiesis in adult rats. Blood 86: 2113-2122.
20. Kobinger, G. P., Weiner, D. J., Yu, Q. C., and Wilson, J. M. (2001). Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo. Nat. Biotechnol. 19: 225-230.
21. Mochizuki, H., Schwartz, J. P., Tanaka, K., Brady, R. O., and Reiser, J. (1998). High-titer human immunodeficiency virus type 1-based vector systems for gene delivery into non-dividing cells. J. Virol. 72: 8873-8883.
22. Burns, J. C., Friedmann, T., Driever, W., Burrascano, M., and Yee, J. K. (1993). Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells. Proc. Natl. Acad. Sci. USA 90: 8033-8037.
23. Desmaris, N., et al. (2001). Production and neurotropism of lentivirus vectors pseudotyped with lyssavirus envelope glycoproteins. Mol. Ther. 4: 149-156, doi.10.1006/mthe.2001.0431.
24. Watson, D. J., Kobinger, G. P., Passini, M. A., Wilson, J. M., and Wolfe, J. H. (2002). Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, ebola, mokola, LCMV, or MuLV envelope proteins. Mol. Ther. 5: 528-537, doi.10.1006/mthe.2002.05843.
25. Chan, S. Y., Speck, R. F., Ma, M. C., and Goldsmith, M. A. (2000). Distinct mechanisms of entry by envelope glycoproteins of marburg and ebola (zaire) viruses. J. Virol. 74: 4933-4937.
26. Chan, S. Y., et al. (2001). Folate receptor-α is a cofactor for cellular entry by marburg and ebola viruses. Cell 106: 117-126.
27. Kafri, T., Blomer, U., Peterson, D. A., Gage, F. H., and Verma, I. M. (1997). Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat. Genet. 17: 314-317.
28. Trono, D. (2000). Lentiviral vectors: turning a deadly foe into a therapeutic agent. Gene Ther. 7: 20-23.
29. Karpati, G., Gilbert, R., Petrof, B. J., and Nalbantoglu, J. (1997). Gene therapy research for duchenne and becker muscular dystrophies. Curr. Opin. Neurol. 10: 430-435.
30. Park, F., Ohashi, K., and Kay, M. A. (2000). Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver. Blood 96: 1173-1176.
31. Chen, S. J., Tazelaar, J., Moscioni, A. D., and Wilson, J. M. (2000). In vivo selection of hepatocytes transduced with adeno-associated viral vectors. Mol. Ther. 1: 414-422, doi.10.1006/mthe.2000.0065.
32. Hajjar, R. J., del Monte, F., Matsui, T., and Rosenzweig, A. (2000). Prospects for gene therapy for heart failure. Circ. Res. 86: 616-621.
33. Woo, Y. J., et al. (1998). Recombinant adenovirus-mediated cardiac gene transfer of superoxide dismutase and catalase attenuates postischemic contractile dysfunction. Circulation 98: II255-II260.
34. Mason, C. A., et al. (1999). Gene transfer in utero biologically engineers a patent ductus arteriosus in lambs by arresting fibronectin-dependent neointimal formation. Nat. Med. 5: 176-182,
35. Dull, T., et al. (1998). A third-generation lentivirus vector with a conditional packaging system. J. Virol. 72: 8463-8471.
36. Zufferey, R., et al. (1998). Self-inactivating lentivinus vector for safe and efficient in vivo gene delivery. J. Virol. 72: 9873-9880.
37. Follenzi, A., Ailles, L. E., Bakovic, S., Geuna, M., and Naldini, L. (2000). Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat. Genet. 25: 217-222.
38. Niwa, H., Yamamura, K., and Miyazaki, J. (1991). Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 108: 193-199.
39. Aurichhio, A., Hildinger, M., O'Connor, E., Gao, G., and Wilson, J. (2001). Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column. Hum. Gene Ther. 12: 71-76.