Myocardial gene transfer and long-term expression following intracoronary delivery of adeno-associated virus

Kaspar, Brian K.; Roth, David M.; Chin Lai, N.; Drumm, Jeffrey D.; Erickson, Dawn A.; McKirnan, M. Dan; Hammond, H. Kirk

doi:10.1002/jgm.665

Cited by 59Virus-based gene therapy researchRNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

Myocardial gene transfer and long-term expression following intracoronary delivery of adeno-associated virus

Kaspar, Brian K., Roth, David M., Chin Lai, N., Drumm, Jeffrey D., Erickson, Dawn A., McKirnan, M. Dan, Hammond, H. Kirk·2005·The Journal of Gene Medicine

DOI 10.1002/jgm.665

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Abstract

Adeno‐associated viral vectors (AAV) can direct long‐term gene expression in post‐mitotic cells. Previous studies have established that long‐term cardiac gene transfer results from intramuscular injection into the heart. Cardiac gene transfer after direct intracoronary delivery of AAV in vivo, however, has been minimal in degree, and indirect intracoronary delivery, an approach used in an increasing number of studies, appears to be receiving more attention. To determine the utility of indirect intracoronary gene transfer of AAV, we used aortic and pulmonary artery cross clamping followed by proximal aortic injection of AAV encoding enhanced green fluorescent protein (AAV.EGFP) at 10 11 DNase resistant particles (drp; high‐performance liquid chromatography (HPLC)‐purified) per rat. Gene expression was quantified by fluorescent microscopy at four time points up to 1 year after vector delivery, revealing 20–32% transmural gene expression in the left ventricle at each time point. Histological analysis revealed little or no inflammatory response and levels of transgene expression were low in liver and undetectable in lung. In subsequent studies in pigs, direct intracoronary delivery into the left circumflex coronary artery of AAV.EGFP (2.64–5.28 × 10 13 drp; HPLC‐purified) resulted in gene expression in 3 of 4 pigs 8 weeks following injection with no inflammatory response in the heart. PCR analysis confirmed AAV vector presence in the left circumflex perfusion bed. These data indicate that intracoronary delivery of AAV vector is associated with transgene expression in the heart, providing a means to obtain long‐term expression of therapeutic genes. Copyright © 2004 John Wiley & Sons, Ltd.

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Myocardial gene deliveries have been performed in swine with AAVs using methods that deliver vectors preferentially to the myocardium such as myocardial injections, direct intracoronary, and selective retroinfusion coupled with coronary artery occlusion.
“Myocardial gene deliveries have been performed with the aid of AAVs with methods that deliver the vectors preferentially to the myocardium such as myocardial injections (H. Su, et al., 2008), direct intracoronary (Kaspar, et al., 2005), and selective retroinfusion coupled with coronary artery occlusion (Pleger, et al., 2011; Raake, et al., 2008).”
From Small and large animal models in cardiac contraction research: advantages and disadvantages (2014) · cites this paper as Kaspar, et al., 2005

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Authors: Kaspar, B. K., Roth, D. M., Chin Lai, N., Drumm, J. D., Erickson, D. A., McKirnan, M. D., & Hammond, H. K.
Affiliations: Salk Institute for Biological Studies (US)
University of California San Diego (US)
VA San Diego Healthcare System (US)
Corresponding author: Hammond, H. Kirk
ORCID: Kaspar, Brian K.
Roth, David M.
Hammond, H. Kirk
Venue: The Journal of Gene Medicine
Publisher: Wiley
Published: 2004-10-28
Volume: 7
Issue: 3
Pages: 316-324
ISSN: 1099-498X (ISSN-L)
1521-2254
DOI: 10.1002/jgm.665
OpenAlex: W2148002368
Cited by: 59
References: 31
Citations by year: 2023: 1 · 2022: 1 · 2021: 2 · 2018: 1 · 2017: 5 · 2016: 2 · 2015: 2 · 2014: 3 · 2013: 6 · 2012: 8
FWCI: 2.32
Type: journal article
Language: en
Open access: closed
Licence: onlinelibrary.wiley.com/termsAndConditions#vor (vor)
Locations: The Journal of Gene Medicine (publishedVersion)
PubMed (publishedVersion)
Funders: U.S. Department of Veterans Affairs
National Heart, Lung, and Blood Institute
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
Keywords: adeno-associated virus vectors, long-term transgene expression, aortic cross-clamping, coronary perfusion, inflammation, pigs, rats, cardiac gene therapy
MeSH terms: Dependovirus, Animals, Cell Line, Gene Expression Regulation, Genetic Vectors, Heart, Humans, Male, Myocardium, Swine, Tissue Distribution, Rats, Sprague-Dawley, Gene Transfer Techniques, Transgenes, Rats

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Myocardial gene transfer and long-term expression following intracoronary delivery of adeno-associated virus

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