Abstract
Adeno-associated virus (AAV) as a vector for gene therapy of cardiovascular diseases has received much recent attention (1–6). This enthusiasm is based on the success of numerous proof of principal gene transfer experiments. Stable high-level expression has been achieved following a variety of delivery methods (direct cardiac muscle injection, coronary vascular or carotid artery infection) and in a wide range of animal models from rodents to pigs (7–12). A side-by-side comparison between viral and nonviral vectors in rabbit heart suggests that rAAV is much more efficient than nonviral vectors such as naked or liposome-complexed DNA. At the same time, rAAV also displays the least inflammatory response in comparison with other viral vectors such as adenovirus and herpes simplex virus (13). Regulated myocardial transgene expression has also been demonstrated with an AAV vector containing glucocorticoid response elements in rat heart (14).
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References
Lynch, C. M., Aara, P. S., Leonard, J. C., Williams, J. K., Dean, R. H., and Geary, R. L. (1997) Adeno-associated virus vectors for vascular gene delivery. Circ. Res. 80, 497–505.
Marshall, D. J. and Leiden, J. M. (1998) Recent advances in skeletal-muscle-based gene therapy. Curr. Opin. Genet. Dev. 8, 360–365.
Alexander, M. Y., Webster, K. A., McDonald, P. H., and Prentice, H. M. (1999) Gene transfer and models of gene therapy for the myocardium. Clin. Exp. Pharmacol. Physiol. 26, 661–668.
Dedieu, J. F., Mafhoudi, A., Le Roux, A., and Branellec, D. (2000) Vectors for gene therapy of cardiovascular disease. Curr. Cardiol. Rep. 2, 39–47.
Phillips, M. I. (2000) Somatic gene therapy for hypertension. Braz. J. Med. Biol. Res. 33, 715–721.
Phillips, M. I., Galli, S. M., and Mehta, J. L. (2000) The potential role of antisense oligodeoxynucleotide therapy for cardiovascular disease. Drugs 60, 239–248.
Ping, P., Yang, Q., and Hammond, H. K. (1996) Altered beta-adrenergic receptor signaling in heart failure, in vivo gene transfer via adeno and adeno-associated virus. Microcirculation 3, 225–228.
Kaplitt, M. G., Xiao, X., and Samulski, R. J. (1996) Long-term gene transfer in porcine myocardium after coronary infusion of an adeno-associated virus vector. Ann. Thorac. Surg. 62, 1669–1676.
Gnatenko, D., Arnold, T. E., Zololukhins, S., Nuovo, G. J., Muzyczka, N., and Bahou, W. F. (1997) Characterization of recombinant adeno-associated virus-2 as a vehicle for gene delivery and expression into vascular cells. J. Investig. Med. 45, 87–98.
Arnold, T. E., Gnatenko, D., and Bahou, W. F. (1997) In vivo gene transfer into rat arterial walls with novel adeno-associated virus vectors. J. Vasc Surg. 25, 347–355.
Maeda, Y., Ikeda, U., Shimpo, M., et al. (1998) Efficient gene transfer into cardiac myocytes using adeno-associated virus (AAV) vectors. J. Mol. Cell Cardiol. 30, 1341–1348.
Svensson, E. C., Marshall, D. J., Woodard, K., et al. (1999) Efficient and stable transduction of cardiomyocytes after intramyocardial injection or intracoronary perfusion with recombinant adeno-associated virus vectors. Circulation 99, 201–205.
Wright, M. J., Wightman, L. M., Lilley, C., et al. (2001) In vivo myocardial gene transfer: optimization, evaluation and direct comparison of gene transfer vectors. Basic Res. Cardiol. 96, 227–236.
Lee, L. Y., Zhou, X., Polce, D. R., et al. (1999) Exogenous control of cardiac gene therapy: evidence of regulated myocardial transgene expression after adenovirus and adeno-associated virus transfer of expression cassettes containing corticos-teroid response element promoters. J. Thorac. Cardiovasc. Surg. 118, 26–24, discussion 34–25.
Su, H., Lu, R., and. Kan, Y. W. (2000) Adeno-associated viral vector-mediated vascular endothelial growth factor gene transfer induces neovascular formation in ischemic heart. Proc. Natl. Acad. Sci. USA 97, 13,801–13,806.
Phillips, M. I. (1997) Antisense inhibition and adeno-associated viral vector delivery for reducing hypertension. Hypertension 29, 177–187.
Kimura, B., Mohuczy, D., Tang, X., and Phillips, M. T. (2001) Attenuation of hypertension and heart hypertrophy by adeno-associated virus delivering angiotensinogen antisense. Hypertension 37, 376–380.
Kawada, T., Sakamoto, A., Nakazawa, M., et al. (2001) Morphological and physiological restorations of hereditary form of dilated cardiomyopathy by somatic gene therapy. Biochem. Biophys. Res. Commun. 284, 431–435.
Dong, J. Y., Fan, P. D., and Frizzell, R. A. (1996) Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum. Gene Ther. 7, 2101–2112.
Duan, D., Sharma, P., Yang, J., et al. (1998) Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long term episomal persistence in muscle. J. Virol. 72, 8568–8577.
Duan, D., Yan, Z., Yue, Y., and Engelhardt, J. F. (1999) Structural analysis of adeno-associated virus transduction intermediates. Virology 261, 8–14.
Yang, J., Zhou, W., Zhang, Y., Zidon, T., Ritchie, T., and Engelhardt, J. F. (1999) Concatamerization of adeno-associated viral circular genomes ocurs through intermolecular recombination. J. Virol. 73, 9468–9477.
Yan, Z., Zhang, Y., Duan, D., and Engelhardt, J. F. (2000) From the Cover: trans-splicing vectors expand the utility of adeno-associated virus for gene therapy. Proc. Natl. Acad. Sci. USA 97, 6716–6721.
Sun, L., Li, J., and Xiao, X. (2000) Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization. Nat. Med. 6, 599–602.
Nakai, H., Storm, T. A., and Kay, M. A. (2000) Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors [see comments]. Nat. Biotechnol. 18, 527–532.
Duan, D., Yue, Y., Yan, Z., and Englehardt, J. F. (2000) A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation. Nat. Med. 6, 595–598.
Duan, D., Yue, Y., and Engelhardt, J. F. (2001) Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a Quantitative Comparison. Mol. Ther. 4, 383–396.
Senapathy, P. and Carter, B. J. (1984) Molecular cloning of adeno-associated virus variant genomes and generation of infectious virus by recombination in mammalian cells. J. Biol. Chem. 259, 4661–4666.
Graham, F. L., Smiley, J., Russell, W. C., and Naim, R., et al. (1977) Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J. Gen. Virol. 36, 59–74.
Louis, N., Evelegh, C., and Graham, F. L. (1997) Cloning and sequencing of the cellular-viral junctions from the human adenovirus type 5 transformed 293 cell line. Virology 233, 423–429.
Ausubel, F. M., et al. (1995) Current Protocols in Molecular Biology. John Wiley, New York.
Samulski, R. J., Chang, L. S., and Shenk, T. (1987) A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J. Virol. 61, 3096–3101.
Xiao, W., Chirmule, N., Berta, S. C., McCullough, B., Gao, G., and Wilson, J. M. (1999) Gene therapy vectors based on adeno-associated virus type 1. J. Virol. 73, 3994–4003.
Duan, D., Yan, Z., Yue, Y., Ding, W., and Englehardt, J. F. (2001) Enhancement of muscle gene delivery with pseudotyped AAV-5 correlates with myoblast differentiation. J. Virol. 75, 7662–7671.
Senapathy, P., Shapiro, M. B., and Harris, N. L. (1990) Splice junctions, branch point sites, and exons: sequence statistics, identification, and applications to genome project. Methods Enzymol. 183, 252–278.
Duan, D., Yue, Y., Yan, Z., Yang, J., and Engelhardt, J. F. (2000) Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J. Clin. Invest. 105, 1573–1587.
Bartlett, J. and Samulski, R. J. (1997) Methods for the construction and propagation of recombinant adeno-associated virus vectors, in Methods in Molecular Medicine, Gene Therapy Protocols (P., Robbins, ed.), Humana, Totowa, NJ, pp. 25–40.
Kotin, R. M., Siniscalco, M., Samulski, R. J., et al. (1990) Site-specific integration by adeno-associated virus. Proc. Natl. Acad. Sci. USA 87, 2211–2215.
Gao, G. P., Qu, G., Faust, L. Z., et al. (1998) High-titer adeno-associated viral vectors from a Rep/Cap cell line and hybrid shuttle virus. Hum. Gene Ther. 9, 2353–2362.
Inoue, N. and Russell, D. W. (1998) Packaging cells based on inducible gene amplification for the production of adeno-associated virus vectors. J. Virol. 72, 7024–7031.
Liu, X. L., Clark, K. R., and Johnson, P. R. (1999) Production of recombinant adeno-associated virus vectors using a packaging cell line and a hybrid recombinant adenovirus. Gene Ther. 6, 293–299.
Xiao, X., Li, J., and Samulski, R. J. (1998) Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol. 72, 2224–2232.
Collaco, R. F., Cao, X., and Trempe, J. P. (1999) A helper virus-free packaging system for recombinant adeno-associated virus vectors. Gene 238, 397–405.
Matsushita, T., Elliger, S., and Elliger, C. (1998) Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther. 5, 938–945.
Li, J., Samulski, R. J., and Xiao, X. (1997) Role for highly regulated rep gene expression in adeno-associated virus vector production. J. Virol. 71, 5236–5243.
Cao, L., Lui, Y., During, M. J., and Xiao, W. (2000) High-titer, wild-type free recombinant adeno-associated virus vector production using intron-containing helper plasmids [In Process Citation]. J. Virol. 74, 11,456–11,463.
Davidson, B. L., Stein, C. S., Heth, J. A., et al. (2000) Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. Proc. Natl. Acad. Sci. USA 97, 3428–3432.
Chao, H., Lui, Y., Robinowitz, J., Li, C., Samulski, R. J., and Walsh, C. E. (2000) Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors. Mol. Ther. 2, 619–623.
Yan, Z., Ritchie, T. C., Duan, D., and Engelhardt, J. F. (2002) Recombinant AAV mediated gene delivery using dual vector heterodimerization. Methods Enzymol. 346, 334–357.
Turnbull, A. E., Skulimowski, A., Smythe, J. A., and Alexander, I. E. (2000) Adeno–associated virus vectors show variable dependence on divalent cations for thermostability: implications for purification and handling. Hum. Gene Ther. 11, 629–635.
Zolotukhin, S., Byrne, B. J., Mason, E., et al. (1999) Recombinant adeno–associated virus purification using novel methods improves infectious titer and yield. Gene Ther. 6, 973–985.
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.
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Duan, D., Yue, Y., Engelhardt, J.F. (2003). Dual Vector Expansion of the Recombinant AAV Packaging Capacity. In: Metzger, J.M. (eds) Cardiac Cell and Gene Transfer. Methods in Molecular Biology, vol 219. Springer, Totowa, NJ. https://doi.org/10.1385/1-59259-350-X:29
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DOI: https://doi.org/10.1385/1-59259-350-X:29
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