Comparison of adeno-associated virus serotypes and delivery methods for cardiac gene transfer

Human Gene Therapy Methods

Volumn 23, Issue 4, 2012, Pages 234-241

  Fang, Hongfei ^a,b   Lai, Ngai Chin ^a,b   Gao, Mei Hua ^a,b   Miyanohara, Atsushi ^a   Roth, David M ^b,c   Tang, Tong ^a,b   Kirk Hammond, H ^a,b  

^a UNIVERSITY OF CALIFORNIA SAN DIEGO   (United States)

^b VA SAN DIEGO HEALTHCARE SYSTEM   (United States)

^c University of California   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ENHANCED GREEN FLUORESCENT PROTEIN; PARVOVIRUS VECTOR; VIRUS DNA; GREEN FLUORESCENT PROTEIN;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; FLUORESCENCE ANALYSIS; GENE DOSAGE; INTRACORONARY INFUSION; INTRAVENOUS DRUG ADMINISTRATION; MALE; MOUSE; NONHUMAN; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; PROTEIN CONTENT; QUANTITATIVE ANALYSIS; SEROTYPE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS GENOME; WESTERN BLOTTING; ANIMAL; C57BL MOUSE; COMPARATIVE STUDY; GENE TRANSFER; GENE VECTOR; GENETICS; HEART MUSCLE; LIVER; METABOLISM; SEROTYPING;

ANIMALS; DEPENDOVIRUS; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; INJECTIONS, INTRAVENOUS; LIVER; MALE; MICE; MICE, INBRED C57BL; MYOCARDIUM; SEROTYPING;

EID: 84872257415     PISSN: 19466536     EISSN: 19466544     Source Type: Journal    
DOI: 10.1089/hgtb.2012.105     Document Type: Article

References (36)

1. Bish, L.T., Sleeper, M.M., Brainard, B., et al. (2008). Percutaneous transendocardial delivery of self-complementary adenoassociated virus 6 achieves global cardiac gene transfer in canines. Mol. Ther. 16, 1953-1959.
2. Bostick, B., Ghosh, A., Yue, Y., et al. (2007). Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration. Gene Ther. 14, 1605-1609. (Pubitemid 350056232)
3. Davis, J., Westfall, M.V., Townsend, D., et al. (2008). Designing heart performance by gene transfer. Physiol. Rev. 88, 1567-1651.
4. Donahue, J.K., Heldman, A.W., Fraser, H., et al. (2000). Focal modification of electrical conduction in the heart by viral gene transfer. Nat. Med. 6, 1395-1398. (Pubitemid 32001025)
5. Fleury, S., Simeoni, E., Zuppinger, C., et al. (2003). Multiply attenuated, self-inactivating lentiviral vectors efficiently deliver and express genes for extended periods of time in adult rat cardiomyocytes in vivo. Circulation 107, 2375-2382. (Pubitemid 36578511)
6. Flotte, T.R. (2005). Recent developments in recombinant AAVmediated gene therapy for lung diseases. Curr. Gene Ther. 5, 361-366. (Pubitemid 40895020)
7. Gao, G., Qu, G., Burnham, M.S., et al. (2000). Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo. Hum. Gene Ther. 11, 2079-2091.
8. Gregorevic, P., Blankinship, M.J., Allen, J.M., et al. (2004). Systemic delivery of genes to striated muscles using adenoassociated viral vectors. Nat. Med. 10, 828-834. (Pubitemid 39070856)
9. Hansen, J., Qing, K., Kwon, H.J., et al. (2000). Impaired intracellular trafficking of adeno-associated virus type 2 vectors limits efficient transduction of murine fibroblasts. J. Virol. 74, 992-996. (Pubitemid 30036236)
10. Inagaki, K., Fuess, S., Storm, T.A., 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. (Pubitemid 43899488)
11. Iwatate, M., Gu, Y., Dieterle, T., et al. (2003). In vivo highefficiency transcoronary gene delivery and Cre-LoxP gene switching in the adult mouse heart. Gene Ther. 10, 1814-1820. (Pubitemid 37185139)
12. Kaspar, B.K., Roth, D.M., Lai, N.C., et al. (2005). Myocardial gene transfer and long-term expression following intracoronary delivery of adeno-associated virus. J. Gene Med. 7, 316-324. (Pubitemid 40443760)
13. Katz, M.G., Swain, J.D., Tomasulo, C.E., et al. (2011). Current strategies for myocardial gene delivery. J. Mol. Cell. Cardiol. 50, 766-776.
14. Lai, N.C., Roth, D.M., Gao, M.H., et al. (2000). Intracoronary delivery of adenovirus encoding adenylyl cyclase VI increases left ventricular function and cAMP-generating capacity. Circulation 102, 2396-2401.
15. Lai, N.C., Roth, D.M., Gao, M.H., et al. (2004). Intracoronary adenovirus encoding adenylyl cyclase VI increases left ventricular function in heart failure. Circulation 110, 330-336. (Pubitemid 38955502)
16. Lai, N.C., Tang, T., Gao, M.H., et al. (2012). Improved function of the failing rat heart by regulated expression of insulin-like growth factor I via intramuscular gene transfer. Hum. Gene Ther. 23, 255-261.
17. Mandel, R.J. (2010). CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease. Curr. Opin. Mol. Ther. 12, 240-247.
18. Meng, X., Rosenthal, R., and Rubin, D. (1992). Comparing correlated correlation coefficients. Psychol. Bull. 111, 172-175.
19. Nathwani, A.C., Rosales, C., McIntosh, J., et al. (2011). Long-term safety and efficacy following systemic administration of a selfcomplementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol. Ther. 19, 876-885.
20. Pacak, C.A., and Byrne, B.J. (2011). AAV vectors for cardiac gene transfer: experimental tools and clinical opportunities. Mol. Ther. 19, 1582-1590.
21. Palomeque, J., Chemaly, E.R., Colosi, P., et al. (2007). Efficiency of eight different AAV serotypes in transducing rat myocardium in vivo. Gene Ther. 14, 989-997.
22. Pleger, S.T., Shan, C., Ksienzyk, J., et al. (2011). Cardiac AAV9-S100A1 gene therapy rescues post-ischemic heart failure in a preclinical large animal model. Sci. Transl. Med. 3, 92ra64.
23. Prasad, K.M., Xu, Y., Yang, Z., et al. (2011). Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution. Gene Ther. 18, 43-52.
24. Raake, P.W., Schlegel, P., Ksienzyk, J., et al. (2012). AAV6. betaARKct cardiac gene therapy ameliorates cardiac function and normalizes the catecholaminergic axis in a clinically relevant large animal heart failure model. Eur. Heart J. doi: 10.1093/eurheartj/ehr447.
25. Roberts, W.G., and Palade, G.E. (1995). Increased microvascular permeability and endothelial fenestration induced by vascular endothelial growth factor. J. Cell Sci. 108(Pt 6), 2369-2379.
26. Rolling, F. (2004). Recombinant AAV-mediated gene transfer to the retina: gene therapy perspectives. Gene Ther. 11(Suppl 1), S26-32. (Pubitemid 39359444)
27. Roth, D.M., Lai, N.C., Gao, M.H., et al. (2004a). Indirect intracoronary delivery of adenovirus encoding adenylyl cyclase increases left ventricular contractile function in mice. Am. J. Physiol. Heart Circ. Physiol. 287, H172-177. (Pubitemid 38802350)
28. Roth, D.M., Lai, N.C., Gao, M.H., et al. (2004b). Nitroprusside increases gene transfer associated with intracoronary delivery of adenovirus. Hum. Gene Ther. 15, 989-994. (Pubitemid 39372575)
29. Sands, M.S. (2011). AAV-mediated liver-directed gene therapy. Methods Mol. Biol. 807, 141-157.
30. Tang, T., Lai, N.C., Hammond, H.K., et al. (2010). Adenylyl cyclase 6 deletion reduces LV hypertrophy, dilation, dysfunciton and fibrosis in pressure-overloaded female mice. J. Am. Coll. Cardiol. 55, 1476-1486.
31. Tilemann, L., Ishikawa, K., Weber, T., and Hajjar, R.J. (2012). Gene therapy for heart failure. Circ. Res. 110, 777-793.
32. Vandenberghe, L.H., Wilson, J.M., and Gao, G. (2009). Tailoring the AAV vector capsid for gene therapy. Gene Ther. 16, 311-319.
33. White, J.D., Thesier, D.M., Swain, J.B., et al. (2011). Myocardial gene delivery using molecular cardiac surgery with recombinant adeno-associated virus vectors in vivo. Gene Ther. 18, 546-552.
34. Zincarelli, C., Soltys, S., Rengo, G., and Rabinowitz, J.E. (2008). Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol. Ther. 16, 1073-1080. (Pubitemid 351737072)
35. Zincarelli, C., Soltys, S., Rengo, G., et al. (2010). Comparative cardiac gene delivery of adeno-associated virus serotypes 1-9 reveals that AAV6 mediates the most efficient transduction in mouse heart. Clin. Transl. Sci. 3, 81-89.
36. Zolotukhin, S., Potter, M., Zolotukhin, I., et al. (2002). Production and purification of serotype 1, 2, and 5 recombinant adenoassociated viral vectors. Methods 28, 158-167. (Pubitemid 35341079)