Cardiac gene therapy: optimization of gene delivery techniques in vivo.

Human Gene Therapy
Michael G KatzCharles R Bridges

Abstract

Vector-mediated cardiac gene therapy holds tremendous promise as a translatable platform technology for treating many cardiovascular diseases. The ideal technique is one that is efficient and practical, allowing for global cardiac gene expression, while minimizing collateral expression in other organs. Here we survey the available in vivo vector-mediated cardiac gene delivery methods--including transcutaneous, intravascular, intramuscular, and cardiopulmonary bypass techniques--with consideration of the relative merits and deficiencies of each. Review of available techniques suggests that an optimal method for vector-mediated gene delivery to the large animal myocardium would ideally employ retrograde and/or anterograde transcoronary gene delivery,extended vector residence time in the coronary circulation, an increased myocardial transcapillary gradient using physical methods, increased endothelial permeability with pharmacological agents, minimal collateral gene expression by isolation of the cardiac circulation from the systemic, and have low immunogenicity.

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Citations

Jul 14, 2010·Journal of Cardiovascular Translational Research·Henriette Brinks, Walter J Koch
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Jul 21, 2017·Human Gene Therapy. Clinical Development·Michael G KatzCharles R Bridges
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Aug 24, 2019·International Journal of Cardiology·Amar TrivediRishi Arora
May 31, 2021·Journal of Cardiovascular Electrophysiology·Shin YooRishi Arora

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Methods Mentioned

BETA
transfection

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