Browse > Article
http://dx.doi.org/10.4333/KPS.2002.32.3.153

Current Status of Gene Therapy as a New Drug Delivery System  

Bae, Yun-Sung (College of Engineering, Institute of Biotechnology, Department of Bioscience and Biotechnology, Sejong University,Microbiology Laboratory, Quality Control Department, Kyung Dong Pharm. Co. Ltd.)
Cho, Jung-Yoon (College of Engineering, Institute of Biotechnology, Department of Bioscience and Biotechnology, Sejong University)
Ji, Sang-Mi (College of Engineering, Institute of Biotechnology, Department of Bioscience and Biotechnology, Sejong University)
Lee, Young-Joo (College of Engineering, Institute of Biotechnology, Department of Bioscience and Biotechnology, Sejong University)
Publication Information
Journal of Pharmaceutical Investigation / v.32, no.3, 2002 , pp. 153-159 More about this Journal
Abstract
Gene therapy is fundamentally a sophisticated drug delivery technology to cure a disease by the transfer of genetic material to modify living cells. In other words, the gene is used as a therapeutic drug much like a chemical compound is employed in chemotherapy. Currently almost 600 clinical trials are underway worldwide since the first clinical trials carried out in 1990 to treat adenosine deaminase deficiency using retroviral vectors. Despite the great progress still is there no gene therapy product being approved as a new drug. This is partly due to a lack of an ideal gene delivery system that is safe and can provide stable, optimal level production of the therapeutic proteins in the cell. This review covers the current status of several different biological and physico-chemical agents that are being developed as gene delivery vehicles. Although gene therapy promises great hopes toward the cure of a broad spectrum of genetic and acquired diseases, the success of gene therapy heavily asks for the development of vector systems for safe and efficient application in humans.
Keywords
Gene therapy; Gene delivery system; Viral vector; Non-viral vector; Nanospheres;
Citations & Related Records
연도 인용수 순위
  • Reference
1 R. Zufferey, D. Nagy, R.J. Mandel, I.Naldini and D. Trono, Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo, Nat. Biotechnol., 15, 871-875 (1997)   DOI   ScienceOn
2 T. Kafri, U. Blomer, D.A. Peterson, F.H. Gage and I.M. Verma, Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors, Nat. Genet., 17, 314-317 (1997)   DOI   ScienceOn
3 L. Naldini, U, Blomer, F.H. Gage, D, Trono and I.M. Verma, Efficient transfer, integration, and sustained long-term expre- ssion of the transgene in adult rat brains injected with a lentiviral vector, Proc. Natl. Acad. Sci. U.S.A., 93, 11382-11388 (1996)   DOI   ScienceOn
4 E. Tomlinson and A.P. Rolland, Controllable gene therapy Pharmaceutics of non-viral gene delivery systems, J. Control. Rel., 39, 357-372 (1996)   DOI   ScienceOn
5 J.P. Yang and L. Huang, Overcoming the inhibitory effect of serum on lipofection by increasing the charge ratio of cationic liposome to DNA, Gene Ther., 4, 950-960 (1997)   DOI   ScienceOn
6 S.G. Martin amd J.C. Murray, Gene-transfer systems for human endothelial cells, Adv. Drug Deliv. Rev., 41, 223-233 (2000)   DOI   ScienceOn
7 D. Armentano, J. Zabner, C. Sacks, C.C. Sookdeo, M.P. Smith, J.A. George, S.C. Wadsworth, A.E. Smith, R.J. Gregory, Effect of the E4 region on the persistence of transgene expression from adenovirus vectors, J. Virol., 71, 2408-2416 (1997)
8 T. Shenk, Adenoviruses: The viruses and their replication. in Virology (Fields, B. N., et al., eds) 2111-2148, Raven Publi- shers, Philadelphia, (1996)
9 P.C. Ross, M.L. Hensen, R. Supabphol and S.W. Hui, Multilamellar cationic liposomes are efficient vectors for in vitro gene transfer in serum, J. Liposome Res., 8, 499-520 (1998)   DOI   ScienceOn
10 D.V. Schaffer and D.A. Lauffenburger, Optimization of cell surface binding enhances efficiency and specificity of molecular conjugate gene delivery, J. Biol. Chem., 273, 28004-28009 (1998)   DOI   ScienceOn
11 P. Schoen, L. Bijl and J. Wilschut, Efficient encapsulation of plasmid DNA in anionic liposomes by a freeze/thaw-extrusion procedure, J. Liposome Res., 8, 485-497 (1998)   DOI   ScienceOn
12 G.Y. Kao, L.J. Change and T.M. Allen, Use of targeted cationic liposomes in enhanced DNA delivery to cancer cells, Cancer Gene Ther., 3, 250-256 (1996)
13 R. Sipehia and G. Martucci, High-efficiency transformation of human endothelial cells by Apo E-mediated transfection with plasmid DNA, Biochem. Biophys. Res. Commun., 214, 206-211 (1995)   DOI   ScienceOn
14 C.R. Dass, T.L. Walker, M.A. Burton and E.E. Decruz, Enhan- ced anticancer therapy mediated by specialized liposomes, J. Pharm. Pharmacol., 49, 972-975 (1995)
15 V.L. Truong-Le, J.T. August and K.W. Leong, Controlled gene delivery by DNA-gelatin nanospheres, Hum. Gene Ther., 9, 1709-1717 (1998)   DOI   ScienceOn
16 B. Roizman, The function of herpes simplex virus genes: a primer for genetic engineering of novel vectors, Proc. Natl. Acad. Sci. U.S.A., 93, 11307-11312 (1996)   DOI   ScienceOn
17 D.W. Losordo, P.R. Vale and J.M. Isner, Gene therapy for myocardial angiogenesis, Am. Heart J., 138, S132 (1999)   DOI   ScienceOn
18 D.J. Fink, L.R. Sternberg, P.C. Weber, M. Mata, W.F. Goins and J.C. Glorioso, In vivo expression of $\beta$-galactosidase in hippocampal neurons by HSV-mediated gene transfer, Gene Ther., 3, 11-19 (1992)   DOI   ScienceOn
19 P. Yotnda, D.H. Chen, W. Chiu, P.A. Piedra, A. Davis, N.S. Templeton and M.K. Brenner, Bilamellar cationic liposomes protect adenovectors from preexisting humoral immune responses, Mol. Ther., 5, 233-241 (2002)   DOI   ScienceOn
20 I. Baumgartner, A. Pieczek, O. Manor, R. Blair, M. Kearney, K. Walsh and J.M. Isner, Constitutive expression of phVEGF 165 after intramuscular gene transfer promotes collateral vessel development in patients with critical limb ischemia, Circulation, 97, 1114-1123 (1998)   DOI   ScienceOn
21 M. Cavazzana-Calvo, S. Hacein-Bey, G. de Saint Basile, F. Gross, E. Yvon, P. Nusbaum, F. Selz, C. Hue, S. Certain, J.L. Casanova, P. Bousso, F.L. Deist and A. Fischer, Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease, Science, 288, 669 (2000)   DOI
22 S. Li, M.A. Rizzo, S. Bhattacharya and L. Huang, Charac-terization of cationic lipid-protamine-DNA (LPD) complexes for intravenous gene delivery, Gene Ther., 5, 930-937 (1998)   DOI   ScienceOn
23 K.W. Peng, Strategies for targeting therapeutic gene delivery, Mol. Med. Today, 5, 448-458 (1999)   DOI   ScienceOn
24 S. Li, W.C. Tseng, D.B. Stolz, S.P. Wu, S.C. Watkins and L. Huang, Dynamic changes in the characteristics of cationic lipidic vectors after exposure to mouse serum: implications for intravenous lipofection, Gene Ther., 6, 585-594 (1999)   DOI   ScienceOn
25 H.Q. Mao, K. Roy, V.L. Troung-Le, K.A. Janes, K.Y. Lin, Y. Wang, J.T. August and K.W. Leong, Chitosan-DNA nano- particles as gene carriers: synthesis, characterization and transfection efficiency, J. Control. Rel., 70, 399-421 (2001)   DOI   ScienceOn
26 F.C. MacLaughlin, R.J. Mumper, J. Wang, J.M. Tagliaferri, I. Gill, M. Hinchcliffe and A.P. Rolland, Chitosan and depoly- merized chitosan oligomers as condensing carriers for in vivo plasmid delivery, J. Control. Rel., 56, 259-272 (1998)   DOI   ScienceOn
27 J. Smith, Y. Zhang and R. Niven, Toward development of a non-viral gene therapeutic, Adv. Drug Deliver. Rev., 26, 135-150 (1997)   DOI   ScienceOn
28 M. Nishikawa and L. Huang, Nonviral vectors in the new millennium: Delivery barriers in gene transfer, Hum. Gene Ther., 12, 861-870 (2001)   DOI   ScienceOn
29 The Journal of Gene Medicine website (http://www.wiley. co.uk/genetherapy), Wiley
30 K.R. Clark, X. Liu, J.P. McGrath and P.R. Johnson, Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses, Hum. Gene Ther., 10, 1031-1039 (1999)   DOI   ScienceOn
31 G.J. Nabel, E.G. Nabel, Z.Y. Yang, B.A. Fox, G.E. Plautz, X. Gao, L. Hung, S. Shu, D. Gordon and A.E. Chang, Direct gene transfer with DNA-liposome complexes in melanoma: Expression, biological activity, and lack of toxicity in humans, Pro. Natl. Acad. Sci. U.S.A., 90, 11307-11311 (1993)   DOI   ScienceOn
32 A. Pfeifer and I.M. Verma, Gene therapy: Promises and Problems, Annu. Rev. Genomics Hum. Genet., 2, 177-211 (2001)   DOI   ScienceOn
33 W.J. Choi and C.K. Kim, Recent advance and future strategy in gene delivery system, J. Kor. Pharm. Sci., 30, 1-12 (2000)
34 I.M. Verma and N. Somia, Gene therapy-promises, problems and prospects, Nature, 389, 239-242 (1997)   DOI   ScienceOn
35 W.F. Anderson, Human gene therapy, Nature, 392, 25s (1998)   DOI   ScienceOn
36 C. Plank, K. Mechtler, F.C. Szoka and E. Wagner, Activation of the complement system by synthetic DNA complexes: A potential barrier for intravenous gene delivery, Hum. Gene Ther., 7, 1437-1446 (1996)   DOI   ScienceOn
37 N. Chirmule, K. Propert, S. Magosin, Y. Qian, R. Qian and J. Wilson, Immune responses to adenovirus and adeno-associated virus in humans, Gene Ther., 6, 1574-1583 (1999)   DOI   ScienceOn
38 N. Muzyczka, Use of adeno-associated virus as a general transduction vector for mammalian cells, Curr. Top. Microbiol. Immunol., 158, 97-129 (1992)   DOI
39 G.L. Clayman, A.K. el-Naggar, S.M. Lippman, Y.C. Hen- derson, M. Frederick, J.A. Merritt, L.A. Zumstein, T.M. Timmons, T.J. Liu, L. Ginsberg, J.A. Roth, W.K. Hong, P. Bruso and H. Goepfert, Adenovirus-mediated p53 gene transfer in patients with advanced recurrent head and neck squamous cell carcinoma, J. Clin. Oncol., 16, 2221-2232 (1998)   DOI
40 J. Nemunaitis, S.G. Swisher, T. Timmons, D. Connors, M. Mack, L. Doerksen, D. Weill, J. Wait, D.D. Lawrence, B.L. Kemp, F. Fossella, B.S. Glisson, W.K. Hong, F.R. Khuri, J.M. Kurie, J.J. Lee, J.S. Lee, D.M. Nguyen, J.C. Nesbitt, R. Perez-Soler, K.M. Pisters, J.B. Putnam, W.R. Richli, D.M. Shin, G.L. Walsh, J. Merritt and J. Roth, Adenovirus-mediated p53 gene transfer in sequence with cisplatin to tumors of patients with non-small-cell lung cancer, J. Clin. Oncol., 18, 609-622 (2000)   DOI
41 M.G. Kaplitt, P. Leone, R.J. Samulski, X. Xiao, D.W. Pfaff, K.L. O'Malley and M.J. During, Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain, Nature Genet., 8, 148-154 (1994)   DOI   ScienceOn
42 R.M. Blease, K.W. Culver, A.D. Miller, C.S. Carter, T. Fleisher, M. Clerici, G. Shearer, L. Chang, Y. Chiang, P. Tolstoshev, J.J. Greenblatt, S.A. Rosenberg, H. Klein, M. Berger, C.A. Mullen, W.J. Ramsey, L. Muul, R.A. Morgan and W.F. Anderson, T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years, Science, 270, 475-480 (1995)   DOI   ScienceOn
43 J.C. Glorioso, W.F. Goins and D.J. Fink, Herpes simplex virus-based vectors, Semin. Virol., 3, 265-276 (1992)