Percutaneous transendocardial delivery of self-complementary adeno-associated virus 6 achieves global cardiac gene transfer in canines

Molecular Therapy

Volumn 16, Issue 12, 2008, Pages 1953-1959

  Bish, Lawrence T ^a   Sleeper, Meg M ^b   Brainard, Benjamin ^b   Cole, Stephen ^b   Russell, Nicholas ^b   Withnall, Elanor ^b   Arndt, Jason ^b   Reynolds, Caryn ^b   Davison, Ellen ^b   Sanmiguel, Julio ^a   Wu, Di ^a   Gao, Guangping ^c   Wilson, James M ^a   Lee Sweeney, H ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DOG; GENE TRANSFER; GENOME; HEART CATHETERIZATION; HEART MUSCLE; NONHUMAN; ORGAN DISTRIBUTION; PERCUTANEOUS TRANSENDOCARDIAL DELIVERY; SEROTYPE; TRANSPORT KINETICS; VIRAL GENE DELIVERY SYSTEM;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS 6; ADENOVIRIDAE; ANIMALIA;

EID: 56749102970     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2008.202     Document Type: Article

References (50)

1. Bostick, B, Ghosh, A, Yue, Y, Long, C and Duan, D (2007). Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration. Gene Ther 14: 1605-1609.
2. Gregorevic, P, Blankinship, MJ, Allen, JM, Crawford, RW, Meuse, L, Miller, DG et al. (2004). Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med 10: 828-834.
3. Inagaki, K, Fuess, S, Storm, TA, Gibson, GA, McTiernan, CF, Kay, MA et al. (2006). Robust systemic transduction with AAV9 vectors in mice: Efficient global cardiac gene transfer superior to that of AAV8. Mol Ther 14: 45-53.
4. Pacak, CA, Mah, CS, Thattaliyath, BD, Conlon, TJ, Lewis, MA, Cloutier, DE et al. (2006). Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ: Res 99: E3-e9.
5. Wang, Z, Zhu, T, Qiao, C, Zhou, L, Wang, B, Zhang, J et al. (2005). Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat Biotechnol 23: 321-328.
6. Woo, YJ, Zhang, JC, Taylor, MD, Cohen, JF, Hsu, VM and Sweeney, HL (2005). One year transgene expression with adeno-associated virus cardiac gene transfer. Int J Cardiol 100: 421-426.
7. Vandendriessche, T, Thorrez, L, Acosta-Sanchez, A, Petrus, I, Wang, L, Ma, L et al. (2007). Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy. J Thromb Haemost 5: 16-24.
8. Zincarelli, C, Soltys, S, Rengo, G and Rabinowitz, JE (2008), Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 16: 1073-1080.
9. Palomeque, J, Chemaly, ER, Colosi, P, Wellman, JA, Zhou, S, Del Monte, F et al. (2007). Efficiency of eight different AAV serotypes in transducing rat myocardium in vivo. Gene Ther 14: 989-997.
10. Hoshijima, M, Ikeda, Y, Iwanaga, Y, Minamisawa, S, Date, MO, Gu, Y et al. (2002). Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery. Nat Med 8: 864-871.
11. Gao, GP, Alvira, MR, Wang, L, Calcedo, R, Johnston, J and Wilson, JM (2002). Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99: 11854-11859.
12. Choi, VW, Samulski, RJ and McCarty DM (2005). Effects of adeno-associated virus DNA hairpin structure on recombination. J Virol 79: 6801-6807.
13. Gao, GR Lu, Y, Sun, X, Johnston, J, Calcedo, R, Grant, R et al. (2006). High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes. J Virol 80: 6192-6194.
14. McCarty, DM, Fu, H, Monahan, PE, Toulson, CE, Naik, P and Samulski, RJ (2003). Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther 10: 2112-2118.
15. McCarty, DM, Monahan, PE and Samulski, RJ (2001). Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther 8: 1248-1254.
16. Sipo, I, Fechner, H, Pinkert, S, Suckau, L, Wang, X, Weger, S et al. (2007). Differential internalization and nuclear uncoating of self-complementary adeno-associated virus pseudotype vectors as determinants of cardiac cell transduction. Gene Ther 14: 1319-1329.
17. Lamping, KG, Rios, CD, Chun, JA, Coboshi, H, Davidson, BL and Heistad, DD (1997). Intrapericardial administration of adenovirus for gene transfer. Am J Physiol 272: H310-H317.
18. Lazarous, DF, Shou, M, Stiber, JA, Hodge, E, Thirumurti, V, Goncalves, L et al. (1999). Adenoviral-mediated gene transfer induces sustained pericardial VEGF expression in dogs: Effect on myocardial angiogenesis. Cardiovasc Res 44: 294-302.
19. Ferrarini, M, Arsic, N, Recchia, FA, Zentilin, L, Zacchigna, S, Xu, X et al. (2006). Adeno-associated virus-mediated transduction of VEGF165 improves cardiac tissue viability and functional recovery after permanent coronary occlusion in conscious dogs. Circ Res 98: 954-961.
20. Laguens, R, Cabeza Meckert, P, Vera Janavel, G, De Lorenzi, A, Lascano, E, Negroni, J et al. (2004). Cardiomyocyte hyperplasia after plasmid-mediated vascular endothelial growth factor gene transfer in pigs with chronic myocardial ischemia. J Gene Med 6: 222-227.
21. McTieman, CF, Mathier, MA, Zhu, X, Xiao, X, Klein, E, Swan, CH et al. (2007). Myocarditis following adeno-associated viral gene expression of human soluble TNF receptor (TNFRII-Fc) in baboon hearts. Gene Ther 14: 1613-1622.
22. Vera Janavel, G, Crottogini, A, Cabeza Meckert, P, Cuniberti, L, Mele, A, Papouchado, M et al. (2006). Plasmid-mediated VEGF gene transfer induces cardiomyogenesis and reduces myocardial infarct size in sheep. Gene Ther 13: 1133-1142.
23. Su, H, Yeghiazarians, Y, Lee, A, Huang, Y, Arakawa-Hoyt, J, Ye, J et al. (2008). AAV serotype 1 mediates more efficient gene transfer to pig myocardium than AAV serotype 2 and plasmid. J Gene Med 10 33-41.
24. Bridges, CR, Burkman, JM, Malekan, R, Konig, SM, Chen, H, Yarnall, CB et al. (2002). Global cardiac-specific transgene expression using cardiopulmonary bypass with cardiac isolation. Ann Thorac Surg 73; 1939-1946.
25. Bridges, CR, Gopal, K, Holt, DE, Yarnall, C, Cole, S, Anderson, RB et al. (2005). Efficient myocyte gene delivery with complete cardiac surgical isolation in situ. J Thorac Cardiovasc Surg 130: 1364.
26. Davidson, MJ, Jones, JM, Emani, SM, Wilson, KH, Jaggers, J, Koch, WJ et al. (2001). Cardiac gene delivery with cardiopulmonary bypass. Circulation 104:131-133.
27. Jones, JM, Wilson, KH, Koch, WJ and Milano, CA (2002). Adenoviral gene transfer to the heart during cardiopulmonary bypass: Effect of myocardial protection technique on transgene expression. Eur J Cardiothorac Surg 21: 847-852.
28. Oi, K, Davies, WR, Tazelaar, HD, Bailey, KR, Federspeil, MJ, Russell, SJ et al. (2006). Ex vivo hypothermic recirculatory adenoviral gene transfer to the transplanted pig heart. J Gene Med 8: 795-803.
29. Boekstegers, P, von Degenfeld, G, Giehrl, W, Heinrich, D, Hullin, R, Kupatt, C et al. (2000). Myocardial gene transfer by selective pressure-regulated retroinfusion of coronary veins. Gene Ther 7 232-240.
30. Hayase, M, Del Monte, F, Kawase, Y, Macneill, BD, McGregor, J, Yoneyama, R et al. (2005). Catheter-based antegrade intracoronary viral gene delivery with coronary venous blockade. Am J Physiol Heart Circ Physiol 288: H2995-H3000.
31. Kaye, DM, Preovolos, A, Marshall, T, Byrne, M, Hoshijima, M. Hajjar, R et al. (2007). Percutaneous cardiac recirculation-mediated gene transfer of an inhibitory phospholamban peptide reverses advanced heart failure in large animals. J Am Coll Cardiol 50: 253-260.
32. Raake, PW, Hinkel, R, Muller, S, Delker, S, Kreuzpointer, R, Kupatt, C et al. (2007). Cardio-specific long-term gene expression in a porcine model after selective pressure-regulated retroinfusion of adeno-associated viral (AAV) vectors. Gene Ther 15: 12-17.
33. Sasano, T, Kikuchi, K, McDonald, AD, Lai, S and Donahue, JK (2007). Targeted high-efficiency, homogeneous myocardial gene transfer. J Mol Cell Cardiol 42: 954-961.
34. Fuchs, S, Battler, A and Kornowski, R (2007). Catheter-based stem cell and gene therapy for refractory myocardial ischemia. Nat Clin Pract Cardiovasc Med 4 (suppl. 1): 589-595.
35. Fuchs, S, Dib, N, Cohen, BM, Okubagzi, P, Diethrich, EB, Campbell, A et al. (2006). A randomized, double-blind, placebo-controlled, multicenter, pilot study of the safety and feasibility of catheter-based intramyocardial injection of AdVEGF121 in patients with refractory advanced coronary artery disease. Catheter Cardiovasc Interv 68 372-378.
36. Losordo, DW, Vale, PR, Hendel, RC, Milliken, CE, Fortuin, FD, Cummings, N et al. (2002). Phase 1/2 placebo-controlled, double-blind, dose-escalating trial of myocardial vascular endothelial growth factor 2 gene transfer by catheter delivery in patients with chronic myocardial ischemia. Circulation 105: 2012-2018.
37. Vale, PR, Losordo, DW, Milliken, CE, McDonald, MC, Gravelin, LM, Curry, CM et al. (2001). Randomized, single-blind, placebo-controlled pilot study of catheter-based myocardial gene transfer for therapeutic angiogenesis using left ventricular electromechanical mapping in patients with chronic myocardial ischemia. Circulation 103: 2138-2143.
38. Li, JJ, Ueno, H, Pan, Y, Tomita, H, Yamamoto, H, Kanegae, Y et al. (1995). Percutaneous transluminal gene transfer into canine myocardium in vivo by replication-defective adenovirus. Cardiovasc Res 30: 97-105.
39. Baldasseroni, S, Opasich, C, Gorini, M, Lucci, D, Marchionni, N, Marini, M et al. (2002). Left bundle-branch block is associated with increased 1-year sudden and total mortality rate in 5517 outpatients with congestive heart failure: A report from the Italian network on congestive heart failure. Am Heart J 143: 398-405.
40. Gavira, JJ, Perez-Ilzarbe, M, Abizanda, G, Garcia-Rodriguez, A, Orbe, J, Paramo, JA et al. (2006). A comparison between percutaneous and surgical transplantation of autologous skeletal myoblasts in a swine model of chronic myocardial infarction. Cardiovasc Res 71: 744-753.
41. He, KL, Yi, GH, Sherman, W, Zhou, H, Zhang, GP, Gu, A et al. (2005). Autologous skeletal myoblast transplantation improved hemodynamics and left ventricular function in chronic heart failure dogs. J Heart Lung Transplant 24: 1940-1949.
42. Meliga, E, Duckers, HJ, Spencer, R and Serruys, PW (2007). Rationale and interim analysis data from the SEISMIC study. EuroIntervention Supplement 2 (suppl. B): B84-B88.
43. Saeed, M, Lee, R, Martin, A, Weber, O, Krombach, GA, Schalla, S et al. (2004). Transendocardial delivery of extracellular myocardial markers by using combination X-ray/MR fluoroscopic guidance: Feasibility study in dogs. Radiology 231: 689-696.
44. Andino, LM, Conlon, TJ, Porvasnik, SL, Boye, SL, Hauswirth, WW and Lewin, AS (2007). Rapid, widespread transduction of the murine myocardium using self-complementary adeno-associated virus. Genet Vaccines Ther 5: 13.
45. Su, H, Huang, Y, Takagawa, J, Barcena, A, Arakawa-Hoyt, J, Ye, J et al. (2006). AAV serotype-1 mediates early onset of gene expression in mouse hearts and results in better therapeutic effect. Gene Ther 13: 1495-1502.
46. Lai, L, Davison, BB, Veazey, RS, Fisher, KJ and Baskin, GB (2002). A preliminary evaluation of recombinant adeno-associated virus biodistribution in rhesus monkeys after intrahepatic inoculation in utero. Hum Gene Ther 13: 2027-2039.
47. Wang, Z, Allen, JM, Riddell, SR, Gregorevic, P, Storb, R, Tapscott, SJ et al. (2007). Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy. Hum Gene Ther 16: 18-26.
48. Wang, Z, Kuhr, CS, Allen, JM, Blankinship, M, Gregorevic, P, Chamberlain, JS et al. (2007). Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression. Mol Ther 15: 1160-1166.
49. Muller, OJ, Leuchs, B, Pleger, ST, Grimm, D, Franz, WM, Katus, HA et al. (2006). Improved cardiac gene transfer by transcriptional and transductional targeting of adeno-associated viral vectors. Cardiovasc Res 70: 70-78.
50. Marshall, DJ, Palasis, M, Lepore, JJ, and Leiden, JM (2000). Biocompatibility of cardiovascular gene delivery catheters with adenovirus vectors: An important determinant of the efficiency of cardiovascular gene transfer. Mol Ther 1: 423-429.