Abstract
Gene therapy is a promising approach in the treatment of cardiovascular diseases. The vectors available for cardiovascular gene therapy have significantly improved over time. Cardiac tropism is a primary characteristic of an ideal vector along with a long-term expression profile and a minimal risk of cellular immune response. Preclinical and clinical studies have demonstrated that adeno-associated viral (AAV) vectors are one of the most attractive vehicles for gene transfer. AAV has gained great popularity in the last years because of its biological properties and advantages over other viral vector systems. In this chapter we will describe methods for intracardiac delivery of AAV vector in rats.
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References
Ylä-Herttuala S, Baker AH (2017) Cardiovascular gene therapy: past, present, and future. Mol Ther 25:1095–1106
Ishikawa K, Weber T, Hajjar RJ (2018) Human cardiac gene therapy. Circ Res 123:601–613
Katz MG, Fargnoli AS, Kendle AP et al (2016) Gene therapy in cardiac surgery: clinical trials, challenges, and perspectives. Ann Thorac Surg 101:2407–2416
Scimia MC, Gumpert AM, Koch WJ (2014) Cardiovascular gene therapy for myocardial infarction. Expert Opin Biol Ther 14:183–195
Katz MG, Fargnoli AS, Williams RD et al (2013) Gene therapy delivery systems for enhancing viral and nonviral vectors for cardiac diseases: current concepts and future applications. Hum Gene Ther 24:914–927
Hammoudi N, Ishikawa K, Hajjar RJ (2015) Adeno-associated virus-mediated gene therapy in cardiovascular disease. Curr Opin Cardiol 30:228–234
Katz MG, Gubara SM, Hadas Y et al (2020) Effects of genetic transfection on calcium cycling pathways mediated by double-stranded adeno-associated virus in postinfarction remodeling. J Thorac Cardiovasc Surg 159:1809–1819
Katz MG, Fargnoli AS, Weber T et al (2017) Use of adeno-associated virus vector for cardiac gene delivery in large-animal surgical models of heart failure. Hum Gene Ther Clin Dev 28:157–164
Gao G, Vandenberghe LH, Alvira MR et al (2004) Clades of adeno-associated viruses are widely disseminated in human tissues. J Virol 78:6381–6388
Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M et al (2017) AAV5-factor VIII gene transfer in severe hemophilia a. N Engl J Med 377:2519–2530
Keeler AM, Flotte TR (2019) Recombinant adeno-associated virus gene therapy in light of luxturna (and zolgensma and glybera): where are we, and how did we get here? Annu Rev Virol 6:601–621
Inagaki K, Fuess S, Storm TA 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
Asokan A, Samulski RJ (2013) An emerging adeno-associated viral vector pipeline for cardiac gene therapy. Hum Gene Ther 24:906–913
Hudry E, Andres-Mateos E, Lerner EP, Volak A, Cohen O, Hyman BT et al (2018) Efficient gene transfer to the central nervous system by single-stranded Anc80L65. Mol Ther Methods Clin Dev 10:197–209
Katz MG, Hadas Y, Bailey RA et al (2021) Efficient cardiac gene transfer and early-onset expression of a synthetic adeno-associated viral vector, Anc80L65, after intramyocardial administration. J Thorac Cardiovasc Surg. S0022-5223(21)00915-6. https://doi.org/10.1016/j.jtcvs.2021.05.050
Carvalho LS, Xiao R, Wassmer SJ et al (2018) Synthetic adeno-associated viral vector efficiently targets mouse and nonhuman primate retina in vivo. Hum Gene Ther 29:771–784
Katz MG, Fargnoli AS, Gubara SM et al (2019) Surgical and physiological challenges in the development of left and right heart failure in rat models. Heart Fail Rev 24:759–777
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Katz, M.G., Hadas, Y., Vincek, A.S., Shtraizent, N., Schadt, E., Eliyahu, E. (2022). Cardiac Targeted Adeno-Associated Virus Injection in Rats. In: Ishikawa, K. (eds) Cardiac Gene Therapy. Methods in Molecular Biology, vol 2573. Humana, New York, NY. https://doi.org/10.1007/978-1-0716-2707-5_10
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DOI: https://doi.org/10.1007/978-1-0716-2707-5_10
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