The inhibitory effects of anticoagulation on in vivo gene transfer by adeno-associated viral or adenoviral vectors

Molecular Therapy

Volumn 13, Issue 1, 2006, Pages 88-97

  Schuettrumpf, Joerg ^a   Zou, Jianxiang ^a   Zhang, Yi ^b   Schlachterman, Alexander ^a   Liu, Yi Lin ^a   Edmonson, Shyrie ^a   Xiao, Weidong ^a   Arruda, Valder R ^a  

^a HOSPITAL OF THE UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

Adeno associated virus; Adenovirus; Anticoagulant; Coagulation; Gene therapy; Heparin; Hirudin; Tick anticoagulant peptide

Indexed keywords

ADENOVIRUS VECTOR; ALPHA 1 ANTITRYPSIN; ANTICOAGULANT AGENT; ARGATROBAN; BLOOD CLOTTING FACTOR 10A INHIBITOR; BLOOD CLOTTING FACTOR 5 LEIDEN; BLOOD CLOTTING FACTOR 9; ENOXAPARIN; HEPARIN; HIRUDIN; LEPIRUDIN; LOW MOLECULAR WEIGHT HEPARIN; PARVOVIRUS VECTOR; PROCOAGULANT; THROMBIN; THROMBIN INHIBITOR; TICK ANTICOAGULANT PEPTIDE;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTICOAGULATION; ARTICLE; CONTROLLED STUDY; DRUG DOSE REGIMEN; DRUG SAFETY; GENE EXPRESSION; GENE TRANSFER; GENETIC TRANSDUCTION; HEMOPHILIA B; HUMAN; HUMAN CELL; IN VIVO STUDY; MALE; MOUSE; MOUSE STRAIN; NONHUMAN; PROMOTER REGION; SEROTYPE; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

ADENOVIRIDAE; ANIMALS; ANTICOAGULANTS; BLOOD COAGULATION DISORDERS; CELL LINE; DEPENDOVIRUS; FACTOR V; FACTOR XA; GENE DOSAGE; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEPARIN; HEPATOCYTES; HIRUDINS; HUMANS; MICE; MICE, INBRED BALB C; MICE, INBRED C57BL; PEPTIDES; SPECIES SPECIFICITY; THROMBIN; TRANSDUCTION, GENETIC;

ACARI; ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS - 2; ADENOVIRIDAE; ANIMALIA; PARVOVIRUS;

EID: 28844449356     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2005.08.004     Document Type: Article

References (46)

1. J.M. Leiden Human gene therapy: The good, the bad, and the ugly Circ. Res. 86 2000 923-925
2. S. Yla-Herttuala and K. Alitalo Gene transfer as a tool to induce therapeutic vascular growth Nat. Med. 9 2003 694-701
3. N. Muzcyzka Use of adeno-associated virus as a general transduction vector in mammalian cells Curr. Top. Microbiol. Immunol. 158 1992 97-129
4. C.S. Manno et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B Blood 101 2003 2963-2972
5. K.A. High Clinical gene transfer studies for hemophilia B Semin. Thromb. Hemostasis 30 2004 257-267
6. J.A. Wagner et al. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies Hum. Gene Ther. 13 2002 1349-1359
7. H. Stedman J.M. Wilson R. Finke A.L. Kleckner and J. Mendell Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: Alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors Hum. Gene Ther. 11 2000 777-790
8. C. Janson et al. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain Hum. Gene Ther. 13 2002 1391-1412
9. M.J. During M.G. Kaplitt M.B. Stern and D. Eidelberg Subthalamic GAD gene transfer in Parkinson disease patients who are candidates for deep brain stimulation Hum. Gene Ther. 12 2001 1589-1591
10. C. Summerfold and R.J. Samulski Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions J. Virol. 72 1998 1438-1445
11. C. Summerford J.S. Bartlett and R.J. Samulski Alpha V beta 5 integrin: A co-receptor for adeno-associated virus type 2 infection Nat. Med. 5 1999 78-82
12. K. Qing C. Mah J. Hansen S. Zhou V. Dwarki and A. Srivastava Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2 Nat. Med. 5 1999 71-77
13. Y. Kashiwakura et al. Hepatocyte growth factor receptor is a coreceptor for adeno-associated virus type 2 infection J. Virol. 79 2005 609-614
14. R. Pruchnic et al. The use of adeno-associated virus to circumvent the maturation-dependent viral transduction of muscle fibers Hum. Gene Ther. 11 2000 521-536
15. U. Hacker et al. Standard heparin, low molecular weight heparin, low molecular weight heparinoid, and recombinant hirudin differ in their ability to inhibit transduction by recombinant adeno-associated virus type 2 vectors Gene Ther. 8 2001 966-968
16. J.I. Weitz and J. Hirsh New anticoagulant drugs Chest 119 2001 95S-107S
17. J. Hirsh et al. Heparin and low-molecular-weight heparin: Mechanisms of action, pharmacokinetics, dosing, monitoring, efficacy, and safety Chest 119 2001 64S-94S
18. A. Betz G. Vlasuk P. Bergum and S. Krishnaswamy Selective inhibition of the prothrombinase complex: Factor Va alters macromolecular recognition of a tick anticoagulant peptide mutant by factor Xa Biochemistry 36 1997 181-191
19. C.T. Dunwiddie D.E. Smith E.M. Nutt and G.P. Vlasuk Anticoagulant effects of the selective factor XA inhibitors tick anticoagulant peptide and antistasin in the APTT assay are determined by the relative rate of prothrombinase inhibition Thromb. Res. 64 1991 787-794
20. F. Mingozzi et al. Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector J. Virol. 76 2002 10497-10502
21. S. Momi M. Nasimi M. Colucci G.G. Nenci and P. Gresele Low molecular weight heparins prevent thrombin-induced thrombo-embolism in mice despite low anti-thrombin activity: Evidence that the inhibition of feed-back activation of thrombin generation confers safety advantages over direct thrombin inhibition Haematologica 86 2001 297-302
22. M.S. Aslam S. Sundberg M.N. Sabri D. Cooke and J.B. Lakier Pharmacokinetics of intravenous/subcutaneous Enoxaparin in patients with acute coronary syndrome undergoing percutaneous coronary interventions Catheter. Cardiovasc. Interv. 57 2002 187-190
23. C.H. Miao et al. The kinetics of rAAV integration in the liver Nat. Genet. 19 1998 13-15
24. R.O. Snyder et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors Nat. Genet. 16 1997 270-276
25. G. Gao M. Alvira L. Wang R. Calcedo J. Johnston and J. Wilson Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy Proc. Natl. Acad. Sci. USA 99 2002 11854-11859
26. L. Wang et al. Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8 mediated, liver-directed gene therapy Blood 105 2005 3079-3086
27. G. Di Pasquale et al. Identification of PDGFR as a receptor for AAV-5 transduction Nat. Med. 9 2003 1306-1312
28. T.J. Wickham E.J. Filardo D.A. Cheresh and G.R. Nemerow Integrin alpha v beta 5 selectively promotes adenovirus mediated cell membrane permeabilization J. Cell Biol. 127 1994 257-264
29. J. Walter Q. You J.N. Hagstrom M. Sands and K.A. High Successful expression of human factor IX following repeat administration of adenoviral vector in mice Proc. Natl. Acad. Sci. USA 93 1996 3056-3061
30. J. Kung et al. Human F.IX corrects the bleeding diathesis of mice with hemophilia B Blood 91 1998 784-790
31. D.E. Sabatino et al. Novel hemophilia B mouse models exhibiting a range of mutations in the Factor IX gene Blood 104 2004 2767-2774
32. R. Bertina et al. Mutation in blood coagulation factor V associated with resistance to activated protein C Nature 369 1994 64-67
33. K.G. Mann and M. Kalafatis Factor V: A combination of Dr Jekyll and Mr Hyde Blood 101 2003 20-30
34. J. Cui et al. Spontaneous thrombosis in mice carrying the factor V Leiden mutation Blood 96 2000 4222-4226
35. A.M. Davidoff C.Y. Ng J. Zhou Y. Spence and A.C. Nathwani Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway Blood 102 2003 480-488
36. J.I. Weitz Low-molecular-weight heparins N. Engl. J. Med. 337 1997 688-698
37. M. Le et al. Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy Blood 89 1997 1254-1259
38. S. Coughlin Thrombin signalling and protease-activated receptors Nature 407 2000 258-264
39. B. Hauck L. Chen W. Xiao et al. Generation and characterization of chimeric recombinant AAV vectors Mol. Ther. 7 2003 419-425
40. A. Girod et al. Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2 Nat. Med. 5 1999 1052-1056
41. P. Wu et al. Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism J. Virol. 74 2000 8635-8647
42. W. Shi and J. S. Bartlett RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism Mol. Ther. 7 2003 515-525
43. J.M. Isner Myocardial gene therapy Nature 415 2002 234-239
44. L.V. Tsui et al. Stability of adenoviral vectors following catheter delivery Mol. Ther. 3 2001 122-125
45. D.J. Marshall M. Palasis J.J. Lepore and J.M. Leiden Biocompatibility of cardiovascular gene delivery catheters with adenovirus vectors: An important determinant of the efficiency of cardiovascular gene transfer Mol. Ther. 1 2000 423-429
46. J. Schuettrumpf R.W. Herzog A. Schlachterman A. Kaufhold D.W. Stafford and V.R. Arruda Factor IX variants improve gene therapy efficacy for hemophilia B Blood 105 2005 2316-2323