Development of safe and efficient viral vectors for clinical gene therapy

Journal of Nanoneuroscience

Volumn 1, Issue 1, 2009, Pages 35-41

  Räty, Jani K ^a,b   Lesch, Hanna P ^a,b   Ylä Herttuala, Seppo ^a  

^a UNIVERSITY OF EASTERN FINLAND   (Finland)

^b Finland Ark Therapeutics Oy   (Finland)

Author keywords

Gene therapy; Safety; Vectors; Viruses

Indexed keywords

GENDICINE; ONCORINE; UNCLASSIFIED DRUG; VIRUS VECTOR;

CANCER GENE THERAPY; DRUG EFFECT; DRUG SAFETY; DRUG TARGETING; GENE EXPRESSION; GENE THERAPY; HUMAN; IMMUNE DEFICIENCY; IMMUNOGENICITY; NONHUMAN; REVIEW;

EID: 75249093633     PISSN: 19390637     EISSN: 19390653     Source Type: Journal    
DOI: 10.1166/jns.2009.004     Document Type: Review

References (71)

1. Airenne KJ, Mahonen AJ, Laitinen OH, Yla-Herttuala S (2004) Baculovirus-mediated gene transfer: an evolving new concept.Gene and Cell Therapy (Templeton NS, ed), pp 181-197. Marcel Dekker, Inc., New York, NY.
2. Baekelandt V, Eggermont K, Michiels M, Nuttin B, Debyser Z (2003) Optimized lentiviral vector production and purification procedure prevents immune response after transduction of mouse brain. Gene Ther 10:1933-1940.
3. Bessis N, GarciaCozar FJ, Boissier MC (2004) Immune responses to gene therapy vectors: Influence on vector function and effector mechanisms. Gene Therapy, Suppl 1, 11:S10-S17.
4. Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev P, Greenblatt JJ, Rosenberg SA, Klein H, Berger M, Mullen CA, Ramsey WJ, Muul L, Morgan RA, Anderson WF (1995) T lymphocyte-directed gene therapy for ADASCID: initial trial results after 4 years. Science 270:475-480.
5. Chen L, Woo SL (2007) Correction in female PKU mice by repeated administration of mPAH cDNA using phiBT1 integration system. Mol Ther 15:1789-1795.
6. Chillon M, Lee JH, Fasbender A, Welsh MJ (1998) Adenovirus complexed with polyethylene glycol and cationic lipid is shielded from neutralizing antibodies in vitro. Gene Ther 5:995-1002.
7. Chirmule N, Propert K, Magosin S, Qian Y, Qian R, Wilson J (1999) Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther 6:1574-1583.
8. Choi VW, McCarty DM, Samulski RJ (2005) AAV hybrid serotypes: improved vectors for gene delivery. Curr Gene Ther 5:299-310.
9. Couzin J, Kaiser J (2005) Gene therapy.As Gelsinger case ends, gene therapy suffers another blow. Science 307:1028.
10. Cronin J, Zhang XY, Reiser J (2005) Altering the tropism of lentiviral vectors through pseudotyping. Curr Gene Ther 5:387-398.
11. Donsante A, Miller DG, Li Y, Vogler C, Brunt EM, Russell DW, Sands MS (2007a) AAV vector integration sites in mouse hepatocellular carcinoma. Science 317:477.
12. Donsante A, Miller DG, Li Y, Vogler C, Brunt EM, Russell DW, Sands MS (2007b) AAV vector integration sites in mouse hepatocellular carcinoma. Science 317:477.
13. Edelstein ML, Abedi MR, Wixon J (2007) Gene therapy clinical trials worldwide to 2007-an update. J Gene Med 9:833-842.
14. Ellis J (2005) Silencing and variegation of gammaretrovirus and lentivirus vectors. Hum Gene Ther 16:1241-1246.
15. Ghosh SS, Gopinath P, Ramesh A (2006a) Adenoviral vectors: a promising tool for gene therapy. Appl Biochem Biotechnol 133:9-29.
16. Ghosh SS, Gopinath P, Ramesh A (2006b) Adenoviral vectors: a promising tool for gene therapy. Appl Biochem Biotechnol 133:9-29.
17. Goessler UR, Riedel K, Hormann K, Riedel F (2006) Perspectives of gene therapy in stem cell tissue engineering. Cells Tissues Organs 183:169-179.
18. Grieger JC, Samulski RJ (2005) Adeno-associated virus as a gene therapy vector: vector development, production and clinical applications. Adv Biochem Eng Biotechnol 99:119-145.
19. Hacein-Bey-Abina S, Von KC, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint BG, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, Le DF, Fischer A, Cavazzana-Calvo M (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302:415-419.
20. Halbert CL, Miller AD, McNamara S, Emerson J, Gibson RL, Ramsey B, Aitken ML (2006) Prevalence of neutralizing antibodies against adenoassociated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: implications for gene therapy using AAV vectors. Hum Gene Ther 17:440-447.
21. Harding JA, Engbers CM, Newman MS, Goldstein NI, Zalipsky S (1997) Immunogenicity and pharmacokinetic attributes of poly-(ethylene glycol)-grafted immunoliposomes. Biochim Biophys Acta 1327:181-192.
22. Heyde M, Partridge KA, Oreffo RO, Howdle SM, Shakesheff KM, Garnett MC (2007) Gene therapy used for tissue engineering applications. J Pharm Pharmacol 59:329-350.
23. Immonen A, Vapalahti M, Tyynela K, Hurskainen H, Sandmair A, Vanninen R, Langford G, Murray N, Yla-Herttuala S (2004) AdvHSVtk gene therapy with intravenous ganciclovir improves survival in human malignant glioma: a randomised, controlled study. Mol Ther 10:967-972.
24. Kaiser J (2007a) Clinical research.Death prompts a review of gene therapy vector. Science 317:580.
25. Kaiser J (2007b) Clinical research.Death prompts a review of gene therapy vector. Science 317:580.
26. Kaplitt MG, Feigin A, Tang C, Fitzsimons HL, Mattis P, Lawlor PA, Bland RJ, Young D, Strybing K, Eidelberg D, During MJ (2007) Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369:2097-2105.
27. Koizumi N, Kawabata K, Sakurai F, Watanabe Y, Hayakawa T, Mizuguchi H (2006) Modified adenoviral vectors ablated for coxsackievirus-adenovirus receptor, alphav integrin, and heparan sulfate binding reduce in vivo tissue transduction and toxicity. Hum Gene Ther 17:264-279.
28. Kreppel F, Gackowski J, Schmidt E, Kochanek S (2005) Combined genetic and chemical capsid modifications enable flexible and efficient de- and retargeting of adenovirus vectors. Mol Ther 12:107-117.
29. Lee JW, Bae SH, Jeong JW, Kim SH, Kim KW (2004) Hypoxia-inducible factor (HIF-1)alpha: its protein stability and biological functions. Exp Mol Med 36:1-12.
30. Lee YB, Glover CP, Cosgrave AS, Bienemann A, Uney JB (2005) Optimizing regulatable gene expression using adenoviral vectors. Exp Physiol 90:33-37.
31. Lehtolainen P, Taskinen A, Laukkanen J, Airenne KJ, Heino S, Lappalainen M, Ojala K, Marjomaki V, Martin JF, Kulomaa MS, Yla-Herttuala S (2002) Cloning and characterization of scavidin, a fusion protein for the targeted delivery of biotinylated molecules. J Biol Chem 277:8545-8550.
32. Lehtolainen P, Wirth T, Taskinen AK, Lehenkari P, Leppanen O, Lappalainen M, Pulkkanen K, Marttila A, Marjomaki V, Airenne KJ, Horton M, Kulomaa MS, Yla-Herttuala S (2003) Targeting of biotinylated compounds to its target tissue using a low-density lipoprotein receptor-avidin fusion protein. Gene Therapy 10:2090-2097.
33. Loewen N, Poeschla EM (2005) Lentiviral vectors. Adv Biochem Eng Biotechnol 99:169-191.
34. Magnusson MK, Hong SS, Henning P, Boulanger P, Lindholm L (2002) Genetic retargeting of adenovirus vectors: functionality of targeting ligands and their influence on virus viability. J Gene Med 4:356-370.
35. Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DG, Johnson FA, McClelland A, Scallan C, Skarsgard E, Flake AW, Kay MA, High KA, Glader B (2003) AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101:2963-2972.
36. Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA (2006) Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 12:342-347.
37. Mizuguchi H, Hayakawa T (2004) Targeted adenovirus vectors. Hum Gene Ther 15:1034-1044.
38. Mottershead DG, Alfthan K, Ojala K, Takkinen K, Oker-Blom C (2000) Baculoviral display of functional scFv and synthetic IgG-binding domains. Biochem Biophys Res Commun 275:84-90.
39. Nettelbeck DM, Jerome V, Muller R (1998) A strategy for enhancing the transcriptional activity of weak cell type-specific promoters. Gene Therapy 5:1656-1664.
40. Ogawara K, Rots MG, Kok RJ, Moorlag HE, Van Loenen AM, Meijer DK, Haisma HJ, Molema G (2004) A novel strategy to modify adenovirus tropism and enhance transgene delivery to activated vascular endothelial cells in vitro and in vivo.Hum. Gene Ther. 15:433-443.
41. O'Riordan CR, Lachapelle A, Delgado C, Parkes V, Wadsworth SC, Smith AE, Francis GE (1999) PEGylation of adenovirus with retention of infectivity and protection from neutralizing antibody in vitro and in vivo. Hum Gene Ther 10:1349-1358.
42. Parrott MB, Adams KE, Mercier GT, Mok H, Campos SK, Barry MA (2003) Metabolically biotinylated adenovirus for cell targeting, ligand screening, and vector purification. Mol Ther 8:688-700.
43. Philippe S, Sarkis C, Barkats M, Mammeri H, Ladroue C, Petit C, Mallet J, Serguera C (2006) Lentiviral vectors with a defective integrase allow efficient and sustained transgene expression in vitro and in vivo. Proc Natl Acad Sci USA 103:17684-17689.
44. Pike-Overzet K, de RD, Weerkamp F, Baert MR, Verstegen MM, Brugman MH, Howe SJ, Reinders MJ, Thrasher AJ, Wagemaker G, van Dongen JJ, Staal FJ (2006) Gene therapy: is IL2RG oncogenic in T-cell development? Nature 443:E5-E7.
45. Post DE, Fulci G, Chiocca EA, Van Meir EG (2004) Replicative oncolytic herpes simplex viruses in combination cancer therapies. Curr Gene Ther.4:41-51.
46. Purow B, Staveley-O'Carroll K (2005) Targeting of vaccinia virus using biotin-avidin viral coating and biotinylated antibodies. J Surg Res 123:49-54.
47. Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao GP, Wilson JM, Batshaw ML (2003) Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 80:148-158.
48. Raty JK, Liimatainen T, Kaikkonen MU, Grohn O, Airenne KJ, Yla-Herttuala S (2007) Non-invasive imaging in gene therapy Mol Ther 15:1579-1586.
49. Raty JK, Airenne KJ, Marttila AT, Marjomaki V, Hytonen VP, Lehtolainen P, Laitinen OH, Mahonen AJ, Kulomaa MS, Yla-Herttuala S (2004) Enhanced gene delivery by avidin-displaying baculovirus. Mol Ther 9:282-291.
50. Raty JK, Liimatainen T, Wirth T, Airenne KJ, Ihalainen TO, Huhtala T, Hamerlynck E, Vihinen-Ranta M, Narvanen A, Yla-Herttuala S, Hakumaki JM (2006) Magnetic resonance imaging of viral particle biodistribution in vivo. Gene Therapy 13:1440-1446.
51. Rein DT, Breidenbach M, Curiel DT (2006) Current developments in adenovirus-based cancer gene therapy. Future Oncol 2:137-143.
52. Rodrigues T, Carrondo MJ, Alves PM, Cruz PE (2007) Purification of retroviral vectors for clinical application: biological implications and technological challenges. J Biotechnol 127:520-541.
53. Sadeghi H, Hitt MM (2005) Transcriptionally targeted adenovirus vectors. Curr Gene Ther 5:411-427.
54. Sandmair AM, Loimas S, Puranen P, Immonen A, Kossila M, Puranen M, Hurskainen H, Tyynela K, Turunen M, Vanninen R, Lehtolainen P, Paljarvi L, Johansson R, Vapalahti M, Yla-Herttuala S (2000) Thymidine kinase gene therapy for human malignant glioma, using replication-deficient retroviruses or adenoviruses. Hum Gene Ther 11:2197-2205.
55. Schenk-Braat EA, van Mierlo MM, Wagemaker G, Bangma CH, Kaptein LC (2007) An inventory of shedding data from clinical gene therapy trials. J Gene Med 9:910-921.
56. Tan W, Dong Z, Wilkinson TA, Barbas CF, III, Chow SA (2006) Human immunodeficiency virus type 1 incorporated with fusion proteins consisting of integrase and the designed polydactyl zinc finger protein E2C can bias integration of viral DNA into a predetermined chromosomal region in human cells. J Virol 80:1939-1948.
57. Tenenbaum L, Lehtonen E, Monahan PE (2003) Evaluation of risks related to the use of adeno-associated virus-based vectors. Curr Gene Ther 3:545-565.
58. Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, Otsu M, Sorrentino B, Scobie L, Cameron E, Blyth K, Neil J, Abina SH, Cavazzana-Calvo M, Fischer A (2006) Gene therapy: X-SCID transgene leukaemogenicity. Nature 443:E5-E6.
59. Turunen MP, Puhakka HL, Koponen JK, Hiltunen MO, Rutanen J, Leppanen O, Turunen AM, Narvanen A, Newby AC, Baker AH, Yla-Herttuala S (2002) Peptide-retargeted adenovirus encoding a tissue inhibitor of metalloproteinase-1 decreases restenosis after intravascular gene transfer. Mol Ther 6:306-312.
60. van Dillen, IJ, Mulder NH, Vaalburg W, de Vries EF, Hospers GA (2002) Influence of the bystander effect on HSV-tk/GCV gene therapy. A review. Curr Gene Ther 2:307-322.
61. Vargas J, Jr., Gusella GL, Najfeld V, Klotman ME, Cara A (2004) Novel integrase-defective lentiviral episomal vectors for gene transfer. Hum Gene Ther 15:361-372.
62. Verma IM, Weitzman MD (2005) Gene therapy: twenty-first century medicine. Annu Rev Biochem 74:711-738.
63. Volpers C, Thirion C, Biermann V, Hussmann S, Kewes H, Dunant P, von der MH, Herrmann A, Kochanek S, Lochmuller H (2003) Antibody-mediated targeting of an adenovirus vector modified to contain a synthetic immunoglobulin g-binding domain in the capsid. J Virol 77:2093-2104.
64. Wang CY, Wang S (2006) Astrocytic expression of transgene in the rat brain mediated by baculovirus vectors containing an astrocyte-specific promoter. Gene Ther 13:1447-1456.
65. Woods NB, Bottero V, Schmidt M, Von KC, Verma IM (2006) Gene therapy: therapeutic gene causing lymphoma. Nature 440:1123.
66. Work LM, Nicklin SA, Brain NJ, Dishart KL, Von Seggern DJ, Hallek M, Buning H, Baker AH (2004) Development of efficient viral vectors selective for vascular smooth muscle cells. Mol Ther 9:198-208.
67. Wu Z, Asokan A, Samulski RJ (2006) Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 14:316-327.
68. Yamanaka R (2004) Alphavirus vectors for cancer gene therapy (review). Int J Oncol 24:919-923.
69. Yamashita M and Emerman M (2006) Retroviral infection of nondividing cells: old and new perspectives. V irology 344:88-93.
70. Yi Y, Hahm SH, Lee KH (2005) Retroviral gene therapy: safety issues and possible solutions. Curr Gene Ther 5:25-35.
71. Zaiss AK, Muruve DA (2005) Immune responses to adeno-associated virus vectors. Curr Gene Ther 5:323-331.