Skip to main content

2014 | OriginalPaper | Buchkapitel

2. Viral Gene Therapy Vectors

verfasst von : Yu-Chen Hu

Erschienen in: Gene Therapy for Cartilage and Bone Tissue Engineering

Verlag: Springer Berlin Heidelberg

Aktivieren Sie unsere intelligente Suche, um passende Fachinhalte oder Patente zu finden.

search-config
loading …

Abstract

The vectors for gene delivery can be divided into two classes: viral and nonviral. Despite the rapid progress in the development of nonviral gene delivery, viral vectors such as retrovirus/lentivirus, adenovirus, adeno-associated virus (AAV) and baculovirus mediate more efficient delivery than nonviral vectors, especially for primary cells. This chapter briefly compares the nonviral and viral vectors and mainly discusses the development and characteristics of these viral vectors.

Sie haben noch keine Lizenz? Dann Informieren Sie sich jetzt über unsere Produkte:

Springer Professional "Wirtschaft+Technik"

Online-Abonnement

Mit Springer Professional "Wirtschaft+Technik" erhalten Sie Zugriff auf:

  • über 102.000 Bücher
  • über 537 Zeitschriften

aus folgenden Fachgebieten:

  • Automobil + Motoren
  • Bauwesen + Immobilien
  • Business IT + Informatik
  • Elektrotechnik + Elektronik
  • Energie + Nachhaltigkeit
  • Finance + Banking
  • Management + Führung
  • Marketing + Vertrieb
  • Maschinenbau + Werkstoffe
  • Versicherung + Risiko

Jetzt Wissensvorsprung sichern!

Springer Professional "Technik"

Online-Abonnement

Mit Springer Professional "Technik" erhalten Sie Zugriff auf:

  • über 67.000 Bücher
  • über 390 Zeitschriften

aus folgenden Fachgebieten:

  • Automobil + Motoren
  • Bauwesen + Immobilien
  • Business IT + Informatik
  • Elektrotechnik + Elektronik
  • Energie + Nachhaltigkeit
  • Maschinenbau + Werkstoffe




 

Jetzt Wissensvorsprung sichern!

Literatur
1.
Zurück zum Zitat Wang C-Y, Li F, Yang Y, Guo H-Y, Wu C-X, Wang S (2006) Recombinant baculovirus containing the Diphtheria toxin A gene for malignant glioma therapy. Cancer Res 66:5798–5806 Wang C-Y, Li F, Yang Y, Guo H-Y, Wu C-X, Wang S (2006) Recombinant baculovirus containing the Diphtheria toxin A gene for malignant glioma therapy. Cancer Res 66:5798–5806
2.
Zurück zum Zitat Gao WT, Soloff AC, Lu XH, Montecalvo A, Nguyen DC, Matsuoka Y et al (2006) Protection of mice and poultry from lethal H5N1 avian influenza virus through adenovirus-based immunization. J Virol 80:1959–1964 Gao WT, Soloff AC, Lu XH, Montecalvo A, Nguyen DC, Matsuoka Y et al (2006) Protection of mice and poultry from lethal H5N1 avian influenza virus through adenovirus-based immunization. J Virol 80:1959–1964
3.
Zurück zum Zitat Siltanen A, Kitabayashi K, Patila T, Ono M, Tikkanen I, Sawa Y et al (2011) Bcl-2 improves myoblast sheet therapy in rat chronic heart failure. Tissue Eng Part A 17:115–125 Siltanen A, Kitabayashi K, Patila T, Ono M, Tikkanen I, Sawa Y et al (2011) Bcl-2 improves myoblast sheet therapy in rat chronic heart failure. Tissue Eng Part A 17:115–125
4.
Zurück zum Zitat Siltanen A, Kitabayashi K, Lakkisto P, Makela J, Patila T, Ono M et al (2011) hHGF overexpression in myoblast sheets enhances their angiogenic potential in rat chronic heart failure. PLoS One 6:e19161 Siltanen A, Kitabayashi K, Lakkisto P, Makela J, Patila T, Ono M et al (2011) hHGF overexpression in myoblast sheets enhances their angiogenic potential in rat chronic heart failure. PLoS One 6:e19161
5.
Zurück zum Zitat Rundle CH, Miyakoshi N, Kasukawa Y, Chen ST, Sheng MHC, Wergedal JE et al (2003) In vivo bone formation in fracture repair induced by direct retroviral-based gene therapy with bone morphogenetic protein-4. Bone 32:591–601 Rundle CH, Miyakoshi N, Kasukawa Y, Chen ST, Sheng MHC, Wergedal JE et al (2003) In vivo bone formation in fracture repair induced by direct retroviral-based gene therapy with bone morphogenetic protein-4. Bone 32:591–601
6.
Zurück zum Zitat Evans CH, Ghivizzani SC, Herndon JH, Wasko MC, Reinecke J, Wehling P et al (2000) Clinical trials in the gene therapy of arthritis. Clin Orthop 379:S300–S307 Evans CH, Ghivizzani SC, Herndon JH, Wasko MC, Reinecke J, Wehling P et al (2000) Clinical trials in the gene therapy of arthritis. Clin Orthop 379:S300–S307
7.
Zurück zum Zitat Evans CH, Gouze JN, Gouze E, Robbins PD, Ghivizzani SC (2004) Osteoarthritis gene therapy. Gene Ther 11:379–389 Evans CH, Gouze JN, Gouze E, Robbins PD, Ghivizzani SC (2004) Osteoarthritis gene therapy. Gene Ther 11:379–389
8.
Zurück zum Zitat Ghivizzani SC, Lechman ER, Tio C, Mule KM, Chada S, McCormack JE et al (1997) Direct retrovirus-mediated gene transfer to the synovium of the rabbit knee: implications for arthritis gene therapy. Gene Ther 4:977–982 Ghivizzani SC, Lechman ER, Tio C, Mule KM, Chada S, McCormack JE et al (1997) Direct retrovirus-mediated gene transfer to the synovium of the rabbit knee: implications for arthritis gene therapy. Gene Ther 4:977–982
9.
Zurück zum Zitat Hsu WK, Sugiyama O, Park SH, Conduah A, Feeley BT, Liu NQ et al (2007) Lentiviral-mediated BMP-2 gene transfer enhances healing of segmental femoral defects in rats. Bone 40:931–938 Hsu WK, Sugiyama O, Park SH, Conduah A, Feeley BT, Liu NQ et al (2007) Lentiviral-mediated BMP-2 gene transfer enhances healing of segmental femoral defects in rats. Bone 40:931–938
10.
Zurück zum Zitat Lieberman JR, Ghivizzani SC, Evans CH (2002) Gene transfer approaches to the healing of bone and cartilage. Mol Ther 6:141–147 Lieberman JR, Ghivizzani SC, Evans CH (2002) Gene transfer approaches to the healing of bone and cartilage. Mol Ther 6:141–147
11.
Zurück zum Zitat Cucchiarini M, Madry H, Ma C, Thurn T, Zurakowski D, Menger MD et al (2005) Improved tissue repair in articular cartilage defects in vivo by rAAV-mediated overexpression of human fibroblast growth factor 2. Mol Ther 12:229–238 Cucchiarini M, Madry H, Ma C, Thurn T, Zurakowski D, Menger MD et al (2005) Improved tissue repair in articular cartilage defects in vivo by rAAV-mediated overexpression of human fibroblast growth factor 2. Mol Ther 12:229–238
12.
Zurück zum Zitat Gelse K, Schneider H (2006) Ex vivo gene therapy approaches to cartilage repair. Adv Drug Deliv Rev 58:259–284 Gelse K, Schneider H (2006) Ex vivo gene therapy approaches to cartilage repair. Adv Drug Deliv Rev 58:259–284
13.
Zurück zum Zitat Madry H, Kaul G, Cucchiarini M, Stein U, Zurakowski D, Remberger K et al (2005) Enhanced repair of articular cartilage defects in vivo by transplanted chondrocytes overexpressing insulin-like growth factor I (IGF-I). Gene Ther 12:1171–1179 Madry H, Kaul G, Cucchiarini M, Stein U, Zurakowski D, Remberger K et al (2005) Enhanced repair of articular cartilage defects in vivo by transplanted chondrocytes overexpressing insulin-like growth factor I (IGF-I). Gene Ther 12:1171–1179
14.
Zurück zum Zitat Mierisch CM, Wilson HA, Turner MA, Milbrandt TA, Berthoux L, Hammarskjold ML et al (2003) Chondrocyte transplantation into articular cartilage defects with use of calcium alginate: the fate of the cells. J Bone Joint Surg Am 85A:1757–1767 Mierisch CM, Wilson HA, Turner MA, Milbrandt TA, Berthoux L, Hammarskjold ML et al (2003) Chondrocyte transplantation into articular cartilage defects with use of calcium alginate: the fate of the cells. J Bone Joint Surg Am 85A:1757–1767
15.
Zurück zum Zitat Tsuchiya H, Kitoh H, Sugiura F, Ishiguro N (2003) Chondrogenesis enhanced by overexpression of sox9 gene in mouse bone marrow-derived mesenchymal stem cells. Biochem Biophys Res Commun 301:338–343 Tsuchiya H, Kitoh H, Sugiura F, Ishiguro N (2003) Chondrogenesis enhanced by overexpression of sox9 gene in mouse bone marrow-derived mesenchymal stem cells. Biochem Biophys Res Commun 301:338–343
16.
Zurück zum Zitat Madry H, Zurakowski D, Trippel SB (2001) Overexpression of human insulin-like growth factor-I promotes new tissue formation in an ex vivo model of articular chondrocyte transplantation. Gene Ther 8:1443–1449 Madry H, Zurakowski D, Trippel SB (2001) Overexpression of human insulin-like growth factor-I promotes new tissue formation in an ex vivo model of articular chondrocyte transplantation. Gene Ther 8:1443–1449
17.
Zurück zum Zitat Venkatesan N, Barre L, Benani A, Netter P, Magdalou J, Fournel-Gigleux S et al (2004) Stimulation of proteoglycan synthesis by glucuronosyltransferase-1 gene delivery: a strategy to promote cartilage repair. Proc Natl Acad Sci U S A 101:18087–18092 Venkatesan N, Barre L, Benani A, Netter P, Magdalou J, Fournel-Gigleux S et al (2004) Stimulation of proteoglycan synthesis by glucuronosyltransferase-1 gene delivery: a strategy to promote cartilage repair. Proc Natl Acad Sci U S A 101:18087–18092
18.
Zurück zum Zitat Kimelman-Bleich N, Pelled G, Zilberman Y, Kallai I, Mizrahi O, Tawackoli W et al (2011) Targeted gene-and-host progenitor cell therapy for nonunion bone fracture repair. Mol Ther 19:53–59 Kimelman-Bleich N, Pelled G, Zilberman Y, Kallai I, Mizrahi O, Tawackoli W et al (2011) Targeted gene-and-host progenitor cell therapy for nonunion bone fracture repair. Mol Ther 19:53–59
19.
Zurück zum Zitat Wang YH, Ho ML, Chang JK, Chu HC, Lai SC, Wang GJ (2009) Microporation is a valuable transfection method for gene expression in human adipose tissue-derived stem cells. Mol Ther 17:302–308 Wang YH, Ho ML, Chang JK, Chu HC, Lai SC, Wang GJ (2009) Microporation is a valuable transfection method for gene expression in human adipose tissue-derived stem cells. Mol Ther 17:302–308
20.
Zurück zum Zitat Aslan H, Zilberman Y, Arbeli V, Sheyn D, Matan Y, Liebergall M et al (2006) Nucleofection-based ex vivo nonviral gene delivery to human stem cells as a platform for tissue regeneration. Tissue Eng 12:877–889 Aslan H, Zilberman Y, Arbeli V, Sheyn D, Matan Y, Liebergall M et al (2006) Nucleofection-based ex vivo nonviral gene delivery to human stem cells as a platform for tissue regeneration. Tissue Eng 12:877–889
21.
Zurück zum Zitat Dinser R, Kreppel F, Zaucke F, Blank C, Paulsson M, Kochanek S et al (2001) Comparison of long-term transgene expression after non-viral and adenoviral gene transfer into primary articular chondrocytes. Histochem Cell Biol 116:69–77 Dinser R, Kreppel F, Zaucke F, Blank C, Paulsson M, Kochanek S et al (2001) Comparison of long-term transgene expression after non-viral and adenoviral gene transfer into primary articular chondrocytes. Histochem Cell Biol 116:69–77
22.
Zurück zum Zitat Park JS, Na K, Woo DG, Yang HN, Kim JM, Kim JH et al (2010) Non-viral gene delivery of DNA polyplexed with nanoparticles transfected into human mesenchymal stem cells. Biomaterials 31:124–132 Park JS, Na K, Woo DG, Yang HN, Kim JM, Kim JH et al (2010) Non-viral gene delivery of DNA polyplexed with nanoparticles transfected into human mesenchymal stem cells. Biomaterials 31:124–132
23.
Zurück zum Zitat Kim T-H, Kim M, Eltohamy M, Yun Y-R, Jang J-H, Kim H-W (2013) Efficacy of mesoporous silica nanoparticles in delivering BMP-2 plasmid DNA for in vitro osteogenic stimulation of mesenchymal stem cells. J Biomed Mater Res Part A 101A:1651–1660 Kim T-H, Kim M, Eltohamy M, Yun Y-R, Jang J-H, Kim H-W (2013) Efficacy of mesoporous silica nanoparticles in delivering BMP-2 plasmid DNA for in vitro osteogenic stimulation of mesenchymal stem cells. J Biomed Mater Res Part A 101A:1651–1660
24.
Zurück zum Zitat Thomas CE, Ehrhardt A, Kay MA (2003) Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 4:346–358 Thomas CE, Ehrhardt A, Kay MA (2003) Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 4:346–358
25.
Zurück zum Zitat Somia N, Verma IM (2000) Gene therapy: trials and tribulations. Nat Rev Genet 1:91–99 Somia N, Verma IM (2000) Gene therapy: trials and tribulations. Nat Rev Genet 1:91–99
26.
Zurück zum Zitat Verma IM, Weitzman MD (2005) Gene therapy: twenty-first century medicine. Annu Rev Biochem 74:711–738 Verma IM, Weitzman MD (2005) Gene therapy: twenty-first century medicine. Annu Rev Biochem 74:711–738
27.
Zurück zum Zitat Yang F, Green JJ, Dinio T, Keung L, Cho SW, Park H et al (2009) Gene delivery to human adult and embryonic cell-derived stem cells using biodegradable nanoparticulate polymeric vectors. Gene Ther 16:533–546 Yang F, Green JJ, Dinio T, Keung L, Cho SW, Park H et al (2009) Gene delivery to human adult and embryonic cell-derived stem cells using biodegradable nanoparticulate polymeric vectors. Gene Ther 16:533–546
28.
Zurück zum Zitat Evans CH (2012) Gene delivery to bone. Adv Drug Deliv Rev 64:1331–1340 Evans CH (2012) Gene delivery to bone. Adv Drug Deliv Rev 64:1331–1340
29.
Zurück zum Zitat Lundstrom K (2003) Latest development in viral vectors for gene therapy. Trends Biotechnol 21:117–122 Lundstrom K (2003) Latest development in viral vectors for gene therapy. Trends Biotechnol 21:117–122
30.
Zurück zum Zitat Airenne KJ, Hu Y-C, Kost TA, Smith RH, Kotin RM, Ono C et al (2013) Baculovirus: an insect-derived vector for diverse gene transfer applications. Mol Ther 21:739–749 Airenne KJ, Hu Y-C, Kost TA, Smith RH, Kotin RM, Ono C et al (2013) Baculovirus: an insect-derived vector for diverse gene transfer applications. Mol Ther 21:739–749
31.
Zurück zum Zitat Hu Y-C (2008) Baculoviral vectors for gene delivery: a review. Curr Gene Ther 8:54–65 Hu Y-C (2008) Baculoviral vectors for gene delivery: a review. Curr Gene Ther 8:54–65
32.
Zurück zum Zitat Huang S, Kamihira M (2013) Development of hybrid viral vectors for gene therapy. Biotechnol Adv 31:208–223 Huang S, Kamihira M (2013) Development of hybrid viral vectors for gene therapy. Biotechnol Adv 31:208–223
33.
Zurück zum Zitat Phillips JE, Gersbach CA, Garcia AJ (2007) Virus-based gene therapy strategies for bone regeneration. Biomaterials 28:211–229 Phillips JE, Gersbach CA, Garcia AJ (2007) Virus-based gene therapy strategies for bone regeneration. Biomaterials 28:211–229
34.
Zurück zum Zitat Kofron MD, Laurencin CT (2006) Bone tissue engineering by gene delivery. Adv Drug Deliv Rev 58:555–576 Kofron MD, Laurencin CT (2006) Bone tissue engineering by gene delivery. Adv Drug Deliv Rev 58:555–576
35.
Zurück zum Zitat Kimelman Bleich N, Kallai I, Lieberman JR, Schwarz EM, Pelled G, Gazit D (2012) Gene therapy approaches to regenerating bone. Adv Drug Deliv Rev 64:1320–1330 Kimelman Bleich N, Kallai I, Lieberman JR, Schwarz EM, Pelled G, Gazit D (2012) Gene therapy approaches to regenerating bone. Adv Drug Deliv Rev 64:1320–1330
36.
Zurück zum Zitat Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M et al (1995) T Lymphocyte-directed gene therapy for ADA – SCID: initial trial results after 4 years. Science 270:475–480 Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M et al (1995) T Lymphocyte-directed gene therapy for ADA – SCID: initial trial results after 4 years. Science 270:475–480
37.
Zurück zum Zitat Hirschmann F, Verhoeyen E, Wirth D, Bauwens S, Hauser H, Rudert M (2002) Vital marking of articular chondrocytes by retroviral infection using green fluorescence protein. Osteoarthritis Cartilage 10:109–118 Hirschmann F, Verhoeyen E, Wirth D, Bauwens S, Hauser H, Rudert M (2002) Vital marking of articular chondrocytes by retroviral infection using green fluorescence protein. Osteoarthritis Cartilage 10:109–118
38.
Zurück zum Zitat Yao SY, Sukonnik T, Kean T, Bharadwaj RR, Pasceri P, Ellis J (2004) Retrovirus silencing, variegation, extinction and memory are controlled by a dynamic interplay of multiple epigenetic modifications. Mol Ther 10:27–36 Yao SY, Sukonnik T, Kean T, Bharadwaj RR, Pasceri P, Ellis J (2004) Retrovirus silencing, variegation, extinction and memory are controlled by a dynamic interplay of multiple epigenetic modifications. Mol Ther 10:27–36
39.
Zurück zum Zitat Bandara G, Mueller GM, Galealauri J, Tindal MH, Georgescu HI, Suchanek MK et al (1993) Intraarticular expression of biologically active interleukin-1 receptor antagonist protein by ex vivo gene transfer. Proc Natl Acad Sci U S A 90:10764–10768 Bandara G, Mueller GM, Galealauri J, Tindal MH, Georgescu HI, Suchanek MK et al (1993) Intraarticular expression of biologically active interleukin-1 receptor antagonist protein by ex vivo gene transfer. Proc Natl Acad Sci U S A 90:10764–10768
40.
Zurück zum Zitat Cavazzana-Calvo M, Hacein-Bey S, Basile CD, Gross F, Yvon E, Nusbaum P et al (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288:669–672 Cavazzana-Calvo M, Hacein-Bey S, Basile CD, Gross F, Yvon E, Nusbaum P et al (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288:669–672
41.
Zurück zum Zitat Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCcormack MP, Wulffraat N, Leboulch P et al (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302:415–419 Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCcormack MP, Wulffraat N, Leboulch P et al (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302:415–419
42.
Zurück zum Zitat Fischer A, Cavazzana-Calvo M (2008) Gene therapy of inherited diseases. Lancet 371:2044–2047 Fischer A, Cavazzana-Calvo M (2008) Gene therapy of inherited diseases. Lancet 371:2044–2047
43.
Zurück zum Zitat Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E et al (2008) Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 118:3132–3142 Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E et al (2008) Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 118:3132–3142
44.
Zurück zum Zitat Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC et al (2010) Efficacy of gene therapy for X-linked severe combined immunodeficiency. New Eng J Med 363:355–364 Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC et al (2010) Efficacy of gene therapy for X-linked severe combined immunodeficiency. New Eng J Med 363:355–364
45.
Zurück zum Zitat Spitzer D, Hauser H, Wirth D (1999) Complement-protected amphotropic retroviruses from murine packaging cells. Hum Gene Ther 10:1893–1902 Spitzer D, Hauser H, Wirth D (1999) Complement-protected amphotropic retroviruses from murine packaging cells. Hum Gene Ther 10:1893–1902
46.
Zurück zum Zitat Gouze E, Pawliuk R, Pilapil C, Gouze JN, Fleet C, Palmer GD et al (2002) In vivo gene delivery to synovium by lentiviral vectors. Mol Ther 5:397–404 Gouze E, Pawliuk R, Pilapil C, Gouze JN, Fleet C, Palmer GD et al (2002) In vivo gene delivery to synovium by lentiviral vectors. Mol Ther 5:397–404
47.
Zurück zum Zitat Li Y, Tew SR, Russell AM, Gonzalez KR, Hardingham TE, Hawkins RE (2004) Transduction of passaged human articular chondrocytes with adenoviral, retroviral, and lentiviral vectors and the effects of enhanced expression of SOX9. Tissue Eng 10:575–584 Li Y, Tew SR, Russell AM, Gonzalez KR, Hardingham TE, Hawkins RE (2004) Transduction of passaged human articular chondrocytes with adenoviral, retroviral, and lentiviral vectors and the effects of enhanced expression of SOX9. Tissue Eng 10:575–584
48.
Zurück zum Zitat Yin GY, Liu WM, An P, Li P, Ding I, Planelles V et al (2002) Endostatin gene transfer inhibits joint angiogenesis and pannus formation in inflammatory arthritis. Mol Ther 5:547–554 Yin GY, Liu WM, An P, Li P, Ding I, Planelles V et al (2002) Endostatin gene transfer inhibits joint angiogenesis and pannus formation in inflammatory arthritis. Mol Ther 5:547–554
49.
Zurück zum Zitat Kato K, Miyake K, Igarashi T, Yoshino S, Shimada T (2005) Human immunodeficiency virus vector-mediated intra-articular expression of angiostatin inhibits progression of collagen-induced arthritis in mice. Rheumatol Int 25:522–529 Kato K, Miyake K, Igarashi T, Yoshino S, Shimada T (2005) Human immunodeficiency virus vector-mediated intra-articular expression of angiostatin inhibits progression of collagen-induced arthritis in mice. Rheumatol Int 25:522–529
50.
Zurück zum Zitat 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 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
51.
Zurück zum Zitat Ronen K, Negre O, Roth S, Colomb C, Malani N, Denaro M et al (2011) Distribution of lentiviral vector integration sites in mice following therapeutic gene transfer to treat beta-thalassemia. Mol Ther 19:1273–1286 Ronen K, Negre O, Roth S, Colomb C, Malani N, Denaro M et al (2011) Distribution of lentiviral vector integration sites in mice following therapeutic gene transfer to treat beta-thalassemia. Mol Ther 19:1273–1286
52.
Zurück zum Zitat Heckl D, Schwarzer A, Haemmerle R, Steinemann D, Rudolph C, Skawran B et al (2012) Lentiviral vector induced insertional haploinsufficiency of Ebf1 causes murine leukemia. Mol Ther 20:1187–1195 Heckl D, Schwarzer A, Haemmerle R, Steinemann D, Rudolph C, Skawran B et al (2012) Lentiviral vector induced insertional haploinsufficiency of Ebf1 causes murine leukemia. Mol Ther 20:1187–1195
53.
Zurück zum Zitat Schambach A, Zychlinski D, Ehrnstroem B, Baum C (2013) Biosafety features of lentiviral vectors. Hum Gene Ther 24:132–142 Schambach A, Zychlinski D, Ehrnstroem B, Baum C (2013) Biosafety features of lentiviral vectors. Hum Gene Ther 24:132–142
54.
Zurück zum Zitat Banasik MB, McCray PB (2009) Integrase-defective lentiviral vectors: progress and applications. Gene Ther 17:150–157 Banasik MB, McCray PB (2009) Integrase-defective lentiviral vectors: progress and applications. Gene Ther 17:150–157
55.
Zurück zum Zitat Chick HE, Nowrouzi A, Fronza R, McDonald RA, Kane NM, Alba R et al (2012) Integrase-deficient lentiviral vectors mediate efficient gene transfer to human vascular smooth muscle cells with minimal genotoxic risk. Hum Gene Ther 23:1247–1257 Chick HE, Nowrouzi A, Fronza R, McDonald RA, Kane NM, Alba R et al (2012) Integrase-deficient lentiviral vectors mediate efficient gene transfer to human vascular smooth muscle cells with minimal genotoxic risk. Hum Gene Ther 23:1247–1257
56.
Zurück zum Zitat Imperiale MJ, Kochanek S (2004) Adenovirus vectors: biology, design, and production. Curr Top Microbiol Immunol 273:335–357 Imperiale MJ, Kochanek S (2004) Adenovirus vectors: biology, design, and production. Curr Top Microbiol Immunol 273:335–357
57.
Zurück zum Zitat Roessler BJ, Allen ED, Wilson JM, Hartman JW, Davidson BL (1993) Adenoviral-mediated gene transfer to rabbit synovium in vivo. J Clin Invest 92:1085–1092 Roessler BJ, Allen ED, Wilson JM, Hartman JW, Davidson BL (1993) Adenoviral-mediated gene transfer to rabbit synovium in vivo. J Clin Invest 92:1085–1092
58.
Zurück zum Zitat Yao QP, Wang SJ, Glorioso JC, Evans CH, Robbins PD, Ghivizzani SC et al (2001) Gene transfer of p53 to arthritic joints stimulates synovial apoptosis and inhibits inflammation. Mol Ther 3:901–910 Yao QP, Wang SJ, Glorioso JC, Evans CH, Robbins PD, Ghivizzani SC et al (2001) Gene transfer of p53 to arthritic joints stimulates synovial apoptosis and inhibits inflammation. Mol Ther 3:901–910
59.
Zurück zum Zitat Ghivizzani SC, Lechman ER, Kang R, Tio C, Kolls J, Evans CH et al (1998) Direct adenovirus-mediated gene transfer of interleukin 1 and tumor necrosis factor alpha soluble receptors to rabbit knees with experimental arthritis has local and distal anti-arthritic effects. Proc Natl Acad Sci U S A 95:4613–4618 Ghivizzani SC, Lechman ER, Kang R, Tio C, Kolls J, Evans CH et al (1998) Direct adenovirus-mediated gene transfer of interleukin 1 and tumor necrosis factor alpha soluble receptors to rabbit knees with experimental arthritis has local and distal anti-arthritic effects. Proc Natl Acad Sci U S A 95:4613–4618
60.
Zurück zum Zitat Brower-Toland BD, Saxer RA, Goodrich LR, Mi ZB, Robbins PD, Evans CH et al (2001) Direct adenovirus-mediated insulin-like growth factor I gene transfer enhances transplant chondrocyte function. Hum Gene Ther 12:117–129 Brower-Toland BD, Saxer RA, Goodrich LR, Mi ZB, Robbins PD, Evans CH et al (2001) Direct adenovirus-mediated insulin-like growth factor I gene transfer enhances transplant chondrocyte function. Hum Gene Ther 12:117–129
61.
Zurück zum Zitat Smith P, Shuler FD, Georgescu HI, Ghivizzani SC, Johnstone B, Niyibizi C et al (2000) Genetic enhancement of matrix synthesis by articular chondrocytes-Comparison of different growth factor genes in the presence and absence of interleukin-1. Arthritis Rheum 43:1156–1164 Smith P, Shuler FD, Georgescu HI, Ghivizzani SC, Johnstone B, Niyibizi C et al (2000) Genetic enhancement of matrix synthesis by articular chondrocytes-Comparison of different growth factor genes in the presence and absence of interleukin-1. Arthritis Rheum 43:1156–1164
62.
Zurück zum Zitat Nixon AJ, Haupt JL, Frisbie DD, Morisset SS, McIlwraith CW, Robbins PD et al (2005) Gene-mediated restoration of cartilage matrix by combination insulin-like factor-I/interleukin-1 receptor antagonist therapy. Gene Ther 12:177–186 Nixon AJ, Haupt JL, Frisbie DD, Morisset SS, McIlwraith CW, Robbins PD et al (2005) Gene-mediated restoration of cartilage matrix by combination insulin-like factor-I/interleukin-1 receptor antagonist therapy. Gene Ther 12:177–186
63.
Zurück zum Zitat Marshall E (1999) Gene therapy death prompts review of adenovirus vector. Science 286:2244–2245 Marshall E (1999) Gene therapy death prompts review of adenovirus vector. Science 286:2244–2245
64.
Zurück zum Zitat Kochanek S, Clemens PR, Mitani K, Chen HH, Chan S, Caskey CT (1996) A new adenoviral vector: replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase. Proc Natl Acad Sci U S A 93:5731–5736 Kochanek S, Clemens PR, Mitani K, Chen HH, Chan S, Caskey CT (1996) A new adenoviral vector: replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase. Proc Natl Acad Sci U S A 93:5731–5736
65.
Zurück zum Zitat Evans CH, Gouze E, Gouze JN, Robbins PD, Ghivizzani SC (2006) Gene therapeutic approaches–transfer in vivo. Adv Drug Deliv Rev 58:243–258 Evans CH, Gouze E, Gouze JN, Robbins PD, Ghivizzani SC (2006) Gene therapeutic approaches–transfer in vivo. Adv Drug Deliv Rev 58:243–258
66.
Zurück zum Zitat Meager A (ed) (1999) Gene therapy technologies, applications and regulations. Wiley, New York Meager A (ed) (1999) Gene therapy technologies, applications and regulations. Wiley, New York
67.
Zurück zum Zitat Mingozzi F, High KA (2011) Immune responses to AAV in clinical trials. Curr Gene Ther 11:321–330 Mingozzi F, High KA (2011) Immune responses to AAV in clinical trials. Curr Gene Ther 11:321–330
68.
Zurück zum Zitat Watanabe S, Imagawa T, Boivin GP, Gao GP, Wilson JM, Hirsch R (2000) Adeno-associated virus mediates long-term gene transfer and delivery of chondroprotective IL-4 to murine synovium. Mol Ther 2:147–152 Watanabe S, Imagawa T, Boivin GP, Gao GP, Wilson JM, Hirsch R (2000) Adeno-associated virus mediates long-term gene transfer and delivery of chondroprotective IL-4 to murine synovium. Mol Ther 2:147–152
69.
Zurück zum Zitat Madry H, Cucchiarini M, Terwilliger EF, Trippel SB (2003) Recombinant adeno-associated virus vectors efficiently and persistently transduce chondrocytes in normal and osteoarthritic human articular cartilage. Hum Gene Ther 14:393–402 Madry H, Cucchiarini M, Terwilliger EF, Trippel SB (2003) Recombinant adeno-associated virus vectors efficiently and persistently transduce chondrocytes in normal and osteoarthritic human articular cartilage. Hum Gene Ther 14:393–402
70.
Zurück zum Zitat Ulrich-Vinther M, Stengaard C, Schwarz EM, Goldring MB, Soballe K (2005) Adeno-associated vector mediated gene transfer of transforming growth factor-β1 to normal and osteoarthritic human chondrocytes stimulates cartilage anabolism. Arthritis Rheum 46:2095–2104 Ulrich-Vinther M, Stengaard C, Schwarz EM, Goldring MB, Soballe K (2005) Adeno-associated vector mediated gene transfer of transforming growth factor-β1 to normal and osteoarthritic human chondrocytes stimulates cartilage anabolism. Arthritis Rheum 46:2095–2104
71.
Zurück zum Zitat Yokoo N, Saito T, Uesugi M, Kobayashi N, Xin K-Q, Okuda K et al (2005) Repair of articular cartilage defect by autologous transplantation of basic fibroblast growth factor gene-transduced chondrocytes with adeno-associated virus vector. Arthritis Rheum 52:164–170 Yokoo N, Saito T, Uesugi M, Kobayashi N, Xin K-Q, Okuda K et al (2005) Repair of articular cartilage defect by autologous transplantation of basic fibroblast growth factor gene-transduced chondrocytes with adeno-associated virus vector. Arthritis Rheum 52:164–170
72.
Zurück zum Zitat Ito H, Koefoed M, Tiyapatanaputi P, Gromov K, Goater JJ, Carmouche J et al (2005) Remodeling of cortical bone allografts mediated by adherent rAAV-RANKL and VEGF gene therapy. Nat Med 11:291–297 Ito H, Koefoed M, Tiyapatanaputi P, Gromov K, Goater JJ, Carmouche J et al (2005) Remodeling of cortical bone allografts mediated by adherent rAAV-RANKL and VEGF gene therapy. Nat Med 11:291–297
73.
Zurück zum Zitat Yla-Herttuala S (2012) Endgame: glybera finally recommended for approval as the first gene therapy drug in the European Union. Mol Ther 20:1831–1832 Yla-Herttuala S (2012) Endgame: glybera finally recommended for approval as the first gene therapy drug in the European Union. Mol Ther 20:1831–1832
74.
Zurück zum Zitat Mease PJ, Wei N, Fudman EJ, Kivitz AJ, Schechtman J, Trapp RG et al (2010) Safety, tolerability, and clinical outcomes after intraarticular injection of a recombinant adeno-associated vector containing a tumor necrosis factor antagonist gene: results of a phase 1/2 study. J Rheumatol 37:692–703 Mease PJ, Wei N, Fudman EJ, Kivitz AJ, Schechtman J, Trapp RG et al (2010) Safety, tolerability, and clinical outcomes after intraarticular injection of a recombinant adeno-associated vector containing a tumor necrosis factor antagonist gene: results of a phase 1/2 study. J Rheumatol 37:692–703
75.
Zurück zum Zitat Evans CH, Ghivizzani SC, Robbins PD (2008) Arthritis gene therapy’s first death. Arthritis Res Ther 10:110 Evans CH, Ghivizzani SC, Robbins PD (2008) Arthritis gene therapy’s first death. Arthritis Res Ther 10:110
76.
Zurück zum Zitat Frank KM, Hogarth DK, Miller JL, Mandal S, Mease PJ, Samulski RJ et al (2009) Brief report: investigation of the cause of death in a gene-therapy trial. New Eng J Med 361:161–169 Frank KM, Hogarth DK, Miller JL, Mandal S, Mease PJ, Samulski RJ et al (2009) Brief report: investigation of the cause of death in a gene-therapy trial. New Eng J Med 361:161–169
77.
Zurück zum Zitat Nayak S, Herzog RW (2010) Progress and prospects: immune responses to viral vectors. Gene Ther 17:295–304 Nayak S, Herzog RW (2010) Progress and prospects: immune responses to viral vectors. Gene Ther 17:295–304
78.
Zurück zum Zitat McCarty DM (2008) Self-complementary AAV, vectors; advances and applications. Mol Ther 16:1648–1656MathSciNet McCarty DM (2008) Self-complementary AAV, vectors; advances and applications. Mol Ther 16:1648–1656MathSciNet
79.
Zurück zum Zitat Smith RH, Levy JR, Kotin RM (2009) A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells. Mol Ther 17:1888–1896 Smith RH, Levy JR, Kotin RM (2009) A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells. Mol Ther 17:1888–1896
80.
Zurück zum Zitat Kost TA, Condreay JP (1999) Recombinant baculoviruses as expression vectors for insect and mammalian cells. Curr Opin Biotechnol 10:428–433 Kost TA, Condreay JP (1999) Recombinant baculoviruses as expression vectors for insect and mammalian cells. Curr Opin Biotechnol 10:428–433
81.
Zurück zum Zitat Kost TA, Condreay JP (2002) Recombinant baculoviruses as mammalian cell gene delivery vectors. Trends Biotechnol 20:173–180 Kost TA, Condreay JP (2002) Recombinant baculoviruses as mammalian cell gene delivery vectors. Trends Biotechnol 20:173–180
82.
Zurück zum Zitat Hofmann C, Sandig V, Jennings G, Rudolph M, Schlag P, Strauss M (1995) Efficient gene-transfer into human hepatocytes by baculovirus vectors. Proc Natl Acad Sci U S A 92:10099–10103 Hofmann C, Sandig V, Jennings G, Rudolph M, Schlag P, Strauss M (1995) Efficient gene-transfer into human hepatocytes by baculovirus vectors. Proc Natl Acad Sci U S A 92:10099–10103
83.
Zurück zum Zitat Boyce FM, Bucher NLR (1996) Baculovirus-mediated gene transfer into mammalian cells. Proc Natl Acad Sci U S A 93:2348–2352 Boyce FM, Bucher NLR (1996) Baculovirus-mediated gene transfer into mammalian cells. Proc Natl Acad Sci U S A 93:2348–2352
84.
Zurück zum Zitat Kost TA, Condreay JP, Jarvis DL (2005) Baculovirus as versatile vectors for protein expression in insect and mammalian cells. Nat Biotechnol 23:567–575 Kost TA, Condreay JP, Jarvis DL (2005) Baculovirus as versatile vectors for protein expression in insect and mammalian cells. Nat Biotechnol 23:567–575
85.
Zurück zum Zitat Hu Y-C (2005) Baculovirus as a highly efficient expression vector in insect and mammalian cells. Acta Pharmacol Sin 26:405–416 Hu Y-C (2005) Baculovirus as a highly efficient expression vector in insect and mammalian cells. Acta Pharmacol Sin 26:405–416
86.
Zurück zum Zitat Hu Y-C (2006) Baculovirus vectors for gene therapy. Adv Virus Res 68:287–320 Hu Y-C (2006) Baculovirus vectors for gene therapy. Adv Virus Res 68:287–320
87.
Zurück zum Zitat Ho Y-C, Chen H-C, Wang K-C, Hu Y-C (2004) Highly efficient baculovirus-mediated gene transfer into rat chondrocytes. Biotechnol Bioeng 88:643–651 Ho Y-C, Chen H-C, Wang K-C, Hu Y-C (2004) Highly efficient baculovirus-mediated gene transfer into rat chondrocytes. Biotechnol Bioeng 88:643–651
88.
Zurück zum Zitat Wang K-C, Wu J-C, Chung Y-C, Ho Y-C, Chang MD, Hu Y-C (2005) Baculovirus as a highly efficient gene delivery vector for the expression of hepatitis delta virus antigens in mammalian cells. Biotechnol Bioeng 89:464–473 Wang K-C, Wu J-C, Chung Y-C, Ho Y-C, Chang MD, Hu Y-C (2005) Baculovirus as a highly efficient gene delivery vector for the expression of hepatitis delta virus antigens in mammalian cells. Biotechnol Bioeng 89:464–473
89.
Zurück zum Zitat Merrihew RV, Clay WC, Condreay JP, Witherspoon SM, Dallas WS, Kost TA (2001) Chromosomal integration of transduced recombinant baculovirus DNA in mammalian cells. J Virol 75:903–909 Merrihew RV, Clay WC, Condreay JP, Witherspoon SM, Dallas WS, Kost TA (2001) Chromosomal integration of transduced recombinant baculovirus DNA in mammalian cells. J Virol 75:903–909
90.
Zurück zum Zitat Cheshenko N, Krougliak N, Eisensmith RC, Krougliak VA (2001) A novel system for the production of fully deleted adenovirus vectors that does not require helper adenovirus. Gene Ther 8:846–854 Cheshenko N, Krougliak N, Eisensmith RC, Krougliak VA (2001) A novel system for the production of fully deleted adenovirus vectors that does not require helper adenovirus. Gene Ther 8:846–854
91.
Zurück zum Zitat Jardin BA, Zhao Y, Selvaraj M, Montes J, Tran R, Prakash S et al (2008) Expression of SEAP (secreted alkaline phosphatase) by baculovirus mediated transduction of HEK 293 cells in a hollow fiber bioreactor system. J Biotechnol 135:272–280 Jardin BA, Zhao Y, Selvaraj M, Montes J, Tran R, Prakash S et al (2008) Expression of SEAP (secreted alkaline phosphatase) by baculovirus mediated transduction of HEK 293 cells in a hollow fiber bioreactor system. J Biotechnol 135:272–280
92.
Zurück zum Zitat Liu CY-Y, Chen H-Z, Chao Y-C (2010) Maximizing baculovirus-mediated foreign proteins expression in mammalian cells. Curr Gene Ther 10:232–241 Liu CY-Y, Chen H-Z, Chao Y-C (2010) Maximizing baculovirus-mediated foreign proteins expression in mammalian cells. Curr Gene Ther 10:232–241
93.
Zurück zum Zitat Lesch HP, Laitinen A, Peixoto C, Vicente T, Makkonen KE, Laitinen L et al (2011) Production and purification of lentiviral vectors generated in 293T suspension cells with baculoviral vectors. Gene Ther 18:531–538 Lesch HP, Laitinen A, Peixoto C, Vicente T, Makkonen KE, Laitinen L et al (2011) Production and purification of lentiviral vectors generated in 293T suspension cells with baculoviral vectors. Gene Ther 18:531–538
94.
Zurück zum Zitat Lesch HP, Turpeinen S, Niskanen EA, Mähönen AJ, Airenne KJ, Ylä-Herttuala S (2008) Generation of lentivirus vectors using recombinant baculoviruses. Gene Ther 15:1280–1286 Lesch HP, Turpeinen S, Niskanen EA, Mähönen AJ, Airenne KJ, Ylä-Herttuala S (2008) Generation of lentivirus vectors using recombinant baculoviruses. Gene Ther 15:1280–1286
95.
Zurück zum Zitat Huang K-S, Lo W-H, Chung Y-C, Lai Y-K, Chen C-Y, Chou S-T et al (2007) Combination of baculovirus-mediated gene delivery and packed-bed reactor for scalable production of adeno-associated virus. Hum Gene Ther 18:1161–1170 Huang K-S, Lo W-H, Chung Y-C, Lai Y-K, Chen C-Y, Chou S-T et al (2007) Combination of baculovirus-mediated gene delivery and packed-bed reactor for scalable production of adeno-associated virus. Hum Gene Ther 18:1161–1170
96.
Zurück zum Zitat Matsuo E, Tani H, Lim C, Komoda Y, Okamoto T, Miyamoto H et al (2006) Characterization of HCV-like particles produced in a human hepatoma cell line by a recombinant baculovirus. Biochem Biophys Res Commun 340:200–208 Matsuo E, Tani H, Lim C, Komoda Y, Okamoto T, Miyamoto H et al (2006) Characterization of HCV-like particles produced in a human hepatoma cell line by a recombinant baculovirus. Biochem Biophys Res Commun 340:200–208
97.
Zurück zum Zitat Chen Y-H, Wu J-C, Wang K-C, Chiang Y-W, Lai C-W, Chung Y-C et al (2005) Baculovirus-mediated production of HDV-like particles in BHK cells using a novel oscillating bioreactor. J Biotechnol 118:135–147 Chen Y-H, Wu J-C, Wang K-C, Chiang Y-W, Lai C-W, Chung Y-C et al (2005) Baculovirus-mediated production of HDV-like particles in BHK cells using a novel oscillating bioreactor. J Biotechnol 118:135–147
98.
Zurück zum Zitat Ernst W, Schinko T, Spenger A, Oker-Blom C, Grabherr R (2006) Improving baculovirus transduction of mammalian cells by surface display of a RGD-motif. J Biotechnol 126:237–240 Ernst W, Schinko T, Spenger A, Oker-Blom C, Grabherr R (2006) Improving baculovirus transduction of mammalian cells by surface display of a RGD-motif. J Biotechnol 126:237–240
99.
Zurück zum Zitat Grabherr R, Ernst W (2010) Baculovirus for eukaryotic protein display. Curr Gene Ther 10:195–200 Grabherr R, Ernst W (2010) Baculovirus for eukaryotic protein display. Curr Gene Ther 10:195–200
100.
Zurück zum Zitat Hu Y-C, Yao K, Wu T-Y (2008) Baculovirus as an expression and/or delivery vehicle for vaccine antigens. Expert Rev Vaccines 7:363–371 Hu Y-C, Yao K, Wu T-Y (2008) Baculovirus as an expression and/or delivery vehicle for vaccine antigens. Expert Rev Vaccines 7:363–371
101.
Zurück zum Zitat Madhan S, Prabakaran M, Kwang J (2010) Baculovirus as vaccine vectors. Curr Gene Ther 10:201–213 Madhan S, Prabakaran M, Kwang J (2010) Baculovirus as vaccine vectors. Curr Gene Ther 10:201–213
102.
Zurück zum Zitat Tani H, Abe T, Matsunaga TM, Moiihi K, Matsuura Y (2008) Baculovirus vector for gene delivery and vaccine development. Future Virol 3:35–43 Tani H, Abe T, Matsunaga TM, Moiihi K, Matsuura Y (2008) Baculovirus vector for gene delivery and vaccine development. Future Virol 3:35–43
103.
Zurück zum Zitat Wang S, Balasundaram G (2010) Potential cancer gene therapy by baculoviral transduction. Curr Gene Ther 10:214–225 Wang S, Balasundaram G (2010) Potential cancer gene therapy by baculoviral transduction. Curr Gene Ther 10:214–225
104.
Zurück zum Zitat Condreay JP, Ames RS, Hassan NJ, Kost TA, Merrihew RV, Mossakowska DE et al (2006) Baculoviruses and mammalian cell-based assays for drug screening. Adv Virus Res 68:255–286 Condreay JP, Ames RS, Hassan NJ, Kost TA, Merrihew RV, Mossakowska DE et al (2006) Baculoviruses and mammalian cell-based assays for drug screening. Adv Virus Res 68:255–286
105.
Zurück zum Zitat Condreay JP, Kost TA (2007) Baculovirus expression vectors for insect and mammalian cells. Curr Drug Targets 8:1126–1131 Condreay JP, Kost TA (2007) Baculovirus expression vectors for insect and mammalian cells. Curr Drug Targets 8:1126–1131
106.
Zurück zum Zitat Kost TA, Condreay JP, Ames RS (2010) Baculovirus gene delivery: a flexible assay development tool. Curr Gene Ther 10:168–173 Kost TA, Condreay JP, Ames RS (2010) Baculovirus gene delivery: a flexible assay development tool. Curr Gene Ther 10:168–173
107.
Zurück zum Zitat Sung L-Y, Lo W-H, Chiu H-Y, Chen H-C, Chuang C-K, Lee H-P et al (2007) Modulation of chondrocyte phenotype via baculovirus-mediated growth factor expression. Biomaterials 28:3437–3447 Sung L-Y, Lo W-H, Chiu H-Y, Chen H-C, Chuang C-K, Lee H-P et al (2007) Modulation of chondrocyte phenotype via baculovirus-mediated growth factor expression. Biomaterials 28:3437–3447
108.
Zurück zum Zitat Lo W-H, Hwang S-M, Chuang C-K, Chen C-Y, Hu Y-C (2009) Development of a hybrid baculoviral vector for sustained transgene expression. Mol Ther 17:658–666 Lo W-H, Hwang S-M, Chuang C-K, Chen C-Y, Hu Y-C (2009) Development of a hybrid baculoviral vector for sustained transgene expression. Mol Ther 17:658–666
109.
Zurück zum Zitat Lu C-H, Lin K-J, Chiu H-Y, Chen C-Y, Yen T-C, Hwang S-M et al (2012) Improved chondrogenesis and engineered cartilage formation from TGF-β3-expressing adipose-derived stem cells cultured in the rotating-shaft bioreactor. Tissue Eng Part A 18:2114–2124 Lu C-H, Lin K-J, Chiu H-Y, Chen C-Y, Yen T-C, Hwang S-M et al (2012) Improved chondrogenesis and engineered cartilage formation from TGF-β3-expressing adipose-derived stem cells cultured in the rotating-shaft bioreactor. Tissue Eng Part A 18:2114–2124
110.
Zurück zum Zitat Yeh T-S, Dean Fang Y-H, Lu C-H, Chiu S-C, Yeh C-L, Yen T-C et al (2014) Baculovirus-transduced, VEGF-expressing adipose-derived stem cell sheet for the treatment of myocardium infarction. Biomaterials 35:174–184 Yeh T-S, Dean Fang Y-H, Lu C-H, Chiu S-C, Yeh C-L, Yen T-C et al (2014) Baculovirus-transduced, VEGF-expressing adipose-derived stem cell sheet for the treatment of myocardium infarction. Biomaterials 35:174–184
111.
Zurück zum Zitat Ho Y-C, Lee H-P, Hwang S-M, Lo W-H, Chen H-C, Chung C-K et al (2006) Baculovirus transduction of human mesenchymal stem cell-derived progenitor cells: variation of transgene expression with cellular differentiation states. Gene Ther 13:1471–1479 Ho Y-C, Lee H-P, Hwang S-M, Lo W-H, Chen H-C, Chung C-K et al (2006) Baculovirus transduction of human mesenchymal stem cell-derived progenitor cells: variation of transgene expression with cellular differentiation states. Gene Ther 13:1471–1479
112.
Zurück zum Zitat Lin C-Y, Lu C-H, Luo W-Y, Chang Y-H, Sung L-Y, Chiu H-Y et al (2010) Baculovirus as a gene delivery vector for cartilage and bone tissue engineering. Curr Gene Ther 10:242–254 Lin C-Y, Lu C-H, Luo W-Y, Chang Y-H, Sung L-Y, Chiu H-Y et al (2010) Baculovirus as a gene delivery vector for cartilage and bone tissue engineering. Curr Gene Ther 10:242–254
113.
Zurück zum Zitat Wang C-Y, Wang S (2005) Adeno-associated virus inverted terminal repeats improve neuronal transgene expression mediated by baculoviral vectors in rat brain. Hum Gene Ther 16:1219–1226 Wang C-Y, Wang S (2005) Adeno-associated virus inverted terminal repeats improve neuronal transgene expression mediated by baculoviral vectors in rat brain. Hum Gene Ther 16:1219–1226
114.
Zurück zum Zitat Zeng J, Du J, Zhao Y, Palanisamy N, Wang S (2007) Baculoviral vector-mediated transient and stable transgene expression in human embryonic stem cells. Stem Cells 25:1055–1061 Zeng J, Du J, Zhao Y, Palanisamy N, Wang S (2007) Baculoviral vector-mediated transient and stable transgene expression in human embryonic stem cells. Stem Cells 25:1055–1061
115.
Zurück zum Zitat Chen C-L, Luo W-Y, Lo W-H, Lin K-J, Sung L-Y, Shih Y-S et al (2011) Development of hybrid baculovirus vectors for artificial MicroRNA delivery and prolonged gene suppression. Biotechnol Bioeng 108:2958–2967 Chen C-L, Luo W-Y, Lo W-H, Lin K-J, Sung L-Y, Shih Y-S et al (2011) Development of hybrid baculovirus vectors for artificial MicroRNA delivery and prolonged gene suppression. Biotechnol Bioeng 108:2958–2967
116.
Zurück zum Zitat Luo WY, Shih YS, Hung CL, Lo KW, Chiang CS, Lo WH et al (2012) Development of the hybrid Sleeping Beauty-baculovirus vector for sustained gene expression and cancer therapy. Gene Ther 19:844–851 Luo WY, Shih YS, Hung CL, Lo KW, Chiang CS, Lo WH et al (2012) Development of the hybrid Sleeping Beauty-baculovirus vector for sustained gene expression and cancer therapy. Gene Ther 19:844–851
117.
Zurück zum Zitat Lin C-Y, Lin K-J, Kao C-Y, Chen M-C, Yen T-Z, Lo W-H et al (2011) The role of adipose-derived stem cells engineered with the persistently expressing hybrid baculovirus in the healing of massive bone defects. Biomaterials 32:6505–6514 Lin C-Y, Lin K-J, Kao C-Y, Chen M-C, Yen T-Z, Lo W-H et al (2011) The role of adipose-derived stem cells engineered with the persistently expressing hybrid baculovirus in the healing of massive bone defects. Biomaterials 32:6505–6514
118.
Zurück zum Zitat Lu J, Zhang F, Kay MA (2013) A mini-intronic plasmid (MIP): a novel robust transgene expression vector in vivo and in vitro. Mol Ther 21:954–963 Lu J, Zhang F, Kay MA (2013) A mini-intronic plasmid (MIP): a novel robust transgene expression vector in vivo and in vitro. Mol Ther 21:954–963
119.
Zurück zum Zitat Gracey Maniar LE, Maniar JM, Chen Z-Y, Lu J, Fire AZ, Kay MA (2013) Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level. Mol Ther 21:131–138 Gracey Maniar LE, Maniar JM, Chen Z-Y, Lu J, Fire AZ, Kay MA (2013) Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level. Mol Ther 21:131–138
120.
Zurück zum Zitat Lin C-Y, Chang Y-H, Kao C-Y, Lu C-H, Sung L-Y, Yen T-C et al (2012) Augmented healing of critical-size calvarial defects by baculovirus-engineered MSCs that persistently express growth factors. Biomaterials 33:3682–3692 Lin C-Y, Chang Y-H, Kao C-Y, Lu C-H, Sung L-Y, Yen T-C et al (2012) Augmented healing of critical-size calvarial defects by baculovirus-engineered MSCs that persistently express growth factors. Biomaterials 33:3682–3692
121.
Zurück zum Zitat Raymond CS, Soriano P (2007) High-efficiency FLP and PhiC31 site-specific recombination in mammalian cells. PLoS One 2:e162 Raymond CS, Soriano P (2007) High-efficiency FLP and PhiC31 site-specific recombination in mammalian cells. PLoS One 2:e162
122.
Zurück zum Zitat Turan S, Galla M, Ernst E, Qiao JH, Voelkel C, Schiedlmeier B et al (2011) Recombinase-mediated cassette exchange (RMCE): traditional concepts and current challenges. J Mol Biol 407:193–221 Turan S, Galla M, Ernst E, Qiao JH, Voelkel C, Schiedlmeier B et al (2011) Recombinase-mediated cassette exchange (RMCE): traditional concepts and current challenges. J Mol Biol 407:193–221
123.
Zurück zum Zitat Sung LY, Chen CL, Lin SY, Hwang SM, Lu CH, Li KC et al (2013) Enhanced and prolonged baculovirus-mediated expression by incorporating recombinase system and in cis elements: a comparative study. Nucleic Acids Res 41:e139 Sung LY, Chen CL, Lin SY, Hwang SM, Lu CH, Li KC et al (2013) Enhanced and prolonged baculovirus-mediated expression by incorporating recombinase system and in cis elements: a comparative study. Nucleic Acids Res 41:e139
Metadaten
Titel
Viral Gene Therapy Vectors
verfasst von
Yu-Chen Hu
Copyright-Jahr
2014
Verlag
Springer Berlin Heidelberg
DOI
https://doi.org/10.1007/978-3-642-53923-7_2

Neuer Inhalt