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Current trends of stem cell-mediated gene therapy

줄기 세포 분야의 유전자 치료 연구 동향

  • Oh, Yu-Kyoung (Department of Microbiology and Institute of Medical Research, College of Medicine, Pochon CHA University) ;
  • Chung, Hyung-Min (Cell and Gene Therapy Research Institute, Pochon CHA University)
  • 오유경 (포천 중문 의과대학교 의학과 미생물학 교실) ;
  • 정형민 (포천 중문 의과대학교 세포 유전자 치료 연구소)
  • Published : 2002.06.20

Abstract

Recently, stem cell-mediated gene therapy is emerging as a novel therapeutic approach. For the successful gene modification of stem cells, the development of a suitable gene transfer technique needs to be preceded. This review focuses on the various gene transfer techniques based on nonviral and viral vectors, and physical methods. The advantages and disadvantages of each gene transfer method are compared, and the general properties of these vectors are discussed in relation to the gene transfer in stem cell research. This review also highlights the therapeutic application of stem cell-mediated gene therapy. The choice of gene transfer vectors may vary depending on the type of the stem cells and the target of stem cell therapy. Of various gene transfer methods, viral vector-based gene therapy has been emphasized due to the higher transfection efficiency. The current status and up-to-date findings of stem cell-mediated gene therapy are discussed in the viewpoint of the various targets of stem cell therapy such as the modification of stem cell potency, the acceleration of regeneration process and the formation of expressional organization.

Keywords

References

  1. P.J. Donovan, J. Gearhart, The end of the beginning for pluripotent stem cells. Nature, 414, 92-97 (2001) https://doi.org/10.1038/35102154
  2. J. Nichols, Introducing embryonic stem cells. Curr. Biol., 11, 503-505 (2001) https://doi.org/10.1016/S0960-9822(01)00143-9
  3. R. Lovell Badge, The futhure for stem cell research. Nature, 414, 88-91 (2001) https://doi.org/10.1038/35102150
  4. E.H. Kaji and J.M. Leiden, Gene and stem cell therapies. JAMA., 285, 545-550 (2001) https://doi.org/10.1001/jama.285.5.545
  5. K.E. Matthews and A. Keating, Gene therapy with physical methods of gene transfer. Transfus. Sci., 17, 29-34 (1996) https://doi.org/10.1016/0955-3886(95)00055-0
  6. Y.K. Oh and R. Straubinger, Cellular retention of liposome-delivered compounds modulated by a probenecid-sensitive anion transporter. Pharm. Res., 14, 1203-1209 (1997) https://doi.org/10.1023/A:1012158924547
  7. Y.K. Oh and J.A. Swanson, Different fates of phagocytosed particles following delivery into macrophage lysosomes. J. Cell Biol., 132, 585-593 (1996) https://doi.org/10.1083/jcb.132.4.585
  8. R.I. Mahato, A. Rolland and E. Tomlinson, Cationic lipidbased gene delivery systems: pharmaceutical perspectives. Pharm. Res., 14, 853-859 (1997) https://doi.org/10.1023/A:1012187414126
  9. K.D. Lee, Y.K. Oh, D.A. Portnoy and J.A. Swanson, Delivery of macromolecules into cytosol using liposomes containing hemolysin from Listeria monocytogenes. J. Biol. Chem., 271, 7249-7252 (1996) https://doi.org/10.1074/jbc.271.13.7249
  10. M.S. Hong, S.J. Lim, Y.K. Oh and C.K. Kim, pH-sensitive, serum-stable and long-circulating liposomes as a new drug delivery system. J. Pharm. Pharmacol., 54, 51-58 (2002) https://doi.org/10.1211/0022357021771913
  11. A. Domashenko, S. Gupta and G. Cotsarelis, Efficient delivery of transgenes to human hair follicle progenitor cells using topical lipoplex. Nat. Biotechnol., 18, 420-423 (2000) https://doi.org/10.1038/74480
  12. H. Keller, C. Yunxu, G. Marit, M. Pla, J. Reiffers, J. Theze and P. Froussard, Transgene expression, but not gene delivery, is improved by adhesion-assisted lipofection of hematopoietic cells. Gene Ther., 6, 931-938 (1999) https://doi.org/10.1038/sj.gt.3300896
  13. G. Marit, Y. Cao, P. Froussard, J. Ripoche, M. Dupouy, A. Elandaloussi, F. Lacombe, F.X. Mahon, H. Keller, M. Pla, J. Reiffers and J. Theze, Increased liposome-mediated gene transfer into haematopoietic cells grown in adhesion to stromal or fibroblast cell line monolayers. Eur. J. Haematol., 64, 22-31 (2000) https://doi.org/10.1034/j.1600-0609.2000.80230.x
  14. Y.K. Oh , J.P. Kim , H. Yoon , J.M. Kim , J.S. Yang , C.K. Kim, Prolonged organ retention and safety of plasmid DNA administered in polyethylenimine complexes. Gene Ther., 8, 1587-92 (2001) https://doi.org/10.1038/sj.gt.3301516
  15. Y.K. Oh, D.C. Suh, J.M. Kim, H.G. Choi, K.S. Shin and J.J. Ko, Polyethylenimine-mediated cellular uptake, nucleus trafficking and expression of cytokine plasmid DNA. Gene Ther., in press (2002)
  16. R. Eiges, M. Schuldiner, M. Drukker, O. Yanuka, J. Itskovitz-Eldor and N. Benvenisty, Establishment of human embryonic stem cell-transfected clones carrying a marker for undifferen- tiated cells. Curr. Biol,. 11, 514-518 (2001). https://doi.org/10.1016/S0960-9822(01)00144-0
  17. F.L. Graham, Adenovirus vectors for high-efficiency gene transfer into mammalian cells. Immunol. Today, 21, 426-28 (2000) https://doi.org/10.1016/S0167-5699(00)01676-5
  18. P.E. Monahan and R.J. Samulski, AAV vectors: is clinical success on the horizon? Gene Ther., 7, 24-30 (2000) https://doi.org/10.1038/sj.gt.3301109
  19. G. Daly and Y. Chernajovsky, Recent developments in retroviral-mediated gene transduction. Mol. Ther,. 2, 423-34 (2000) https://doi.org/10.1006/mthe.2000.0211
  20. D. Trono, Lentiviral vectors: turning a deadly foe into a therapeutic agent. Gene Ther., 7, 20-3 (2000) https://doi.org/10.1038/sj.gt.3301105
  21. G.L .Jr. Buchschacher and F. Wong-Staal, Development of lentiviral vectors for gene therapy for human diseases. Blood, 95, 2499-2504 (2000)
  22. N.B. Woods, H. Mikkola, E. Nilsson, K. Olsson, D. Trono, Karlsson, S. Lentiviral-mediated gene transfer into haematopoietic stem cells. J. Intern. Med., 249, 339-343 (2001) https://doi.org/10.1046/j.1365-2796.2001.00806.x
  23. R. Pawliuk, K.A. Westerman, M.E. Fabry, E. Payen, R. Tighe, E.E. Bouhassira, S.A. Acharya, J. Ellis, I.M. London, C.J. Eaves, R.K. Humphries, Y. Beuzard, R.L. Nagel and P. Leboulch, Correction of sickle cell disease in transgenic mouse models by gene therapy. Science, 29, 2368-2371 (2001)
  24. U. Kuhn, A. Terunuma, W. Pfutzner, R.A. Foster and J.C. Vogel, In vivo assessment of gene delivery to keratinocytes by lentiviral vectors. J. Virol., 76, 1496-1504 (2002) https://doi.org/10.1128/JVI.76.3.1496-1504.2002
  25. Y. Cui, J. Golob, E. Kelleher, Z. Ye, D. Pardoll and L. Cheng, Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hemato- poietic stem/progenitor cells. Blood, 99, 399-408 (2002) https://doi.org/10.1182/blood.V99.2.399
  26. E.A. Burton, J.B. Wechuck, S.K. Wendell, W.F. Goins, D.J. Fink and J.C. Glorioso, Multiple applications for replication-defective herpes simplex virus vectors. Stem Cells, 19, 358-377 (2001) https://doi.org/10.1634/stemcells.19-5-358
  27. H. Iwaguro, J. Yamaguchi, C. Kalka, S. Murasawa, H. Masuda, S. Hayashi, Silver M, Li, T, J.M. Isner and Asahara, T. Endothelial progenitor cell vascular endothelial growth factor gene transfer for vascular regeneration. Circulation., 105, 732-738 (2002) https://doi.org/10.1161/hc0602.103673
  28. G. Andsberg, Z. Kokaia, A. Bjorklund, O. Lindvall, and A, Martinez-Serrano, Amelioration of ischaemia-induced neuronal death in the rat striatum by NGF-secreting neural stem cells, Eur. J. Neurosci., 10, 2026-2036 (1998) https://doi.org/10.1046/j.1460-9568.1998.00214.x
  29. T. Asahara, C. Kalka and J.M. Isner, Stem cell therapy and gene transfer for regeneration. Gene Ther., 7, 451-547 (2000) https://doi.org/10.1038/sj.gt.3301142
  30. S.R. Cherry, D. Biniszkiewicz, L. van Parijs, D. Baltimore and R. Jaenisch, Retroviral expression in embryonic stem cells and hematopoietic stem cells. Mol. Cell Biol., 20, 7419-7426 (2000) https://doi.org/10.1128/MCB.20.20.7419-7426.2000
  31. A. Pfeifer, M. Ikawa, Y. Dayn and I.M. Verma, Transgenesis by lentiviral vectors: Lack of gene silencing in mammalian embryonic stem cells and preimplantation embryos. Proc. Natl. Acad. Sci. U. S. A., 99, 2140-2145 (2002) https://doi.org/10.1073/pnas.251682798
  32. B.C. Engel and D.B. Kohn, Stem cell directed gene therapy. Front. Biosci., 426-33( 1999)
  33. M.F. Philips, G. Mattiasson, T. Wieloch, A. Bjorklimd, B.B. Johansson, G. Tomasevic, A. Martinez-Serrano, P.M. Lenzlin-ger, G. Smson, M.S. Grady and T.K. Mackintosh, Neuropro-tective and behavioral efficacy of nerve growth factor-transfected hippocampal progenitor cell transplants after experimental t raumatic brain injury. J. Neurosurg., 94, 765-774 (2001) https://doi.org/10.3171/jns.2001.94.5.0765
  34. G. Turgeman, D.D. Pittman, R. Muller, B.G. Kurkalli, S. Zhou, G. Pelled, A. Peyser, Y. Zilberman, I.K. Moutsatsos and D. Gazit, Engineered human mesenchymal stem cells: a novel platform for skeletal cell mediated gene therapy. J. Gene Med., 3, 240-251 (2001) https://doi.org/10.1002/1521-2254(200105/06)3:3<240::AID-JGM181>3.0.CO;2-A