Heart failure gene therapy: the path to clinical practice

Pleger, Sven T.; Brinks, Henriette; Ritterhoff, Julia; Raake, Philip; Koch, Walter J.; Katus, Hugo A.; Most, Patrick (2013). Heart failure gene therapy: the path to clinical practice. Circulation research, 113(6), pp. 792-809. Lippincott Williams & Wilkins 10.1161/CIRCRESAHA.113.300269

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Gene therapy, aimed at the correction of key pathologies being out of reach for conventional drugs, bears the potential to alter the treatment of cardiovascular diseases radically and thereby of heart failure. Heart failure gene therapy refers to a therapeutic system of targeted drug delivery to the heart that uses formulations of DNA and RNA, whose products determine the therapeutic classification through their biological actions. Among resident cardiac cells, cardiomyocytes have been the therapeutic target of numerous attempts to regenerate systolic and diastolic performance, to reverse remodeling and restore electric stability and metabolism. Although the concept to intervene directly within the genetic and molecular foundation of cardiac cells is simple and elegant, the path to clinical reality has been arduous because of the challenge on delivery technologies and vectors, expression regulation, and complex mechanisms of action of therapeutic gene products. Nonetheless, since the first demonstration of in vivo gene transfer into myocardium, there have been a series of advancements that have driven the evolution of heart failure gene therapy from an experimental tool to the threshold of becoming a viable clinical option. The objective of this review is to discuss the current state of the art in the field and point out inevitable innovations on which the future evolution of heart failure gene therapy into an effective and safe clinical treatment relies.

Item Type:	Journal Article (Review Article)
Division/Institute:	04 Faculty of Medicine > Department of Cardiovascular Disorders (DHGE) > Clinic of Heart Surgery
UniBE Contributor:	Most, Henriette
Subjects:	600 Technology > 610 Medicine & health
ISSN:	0009-7330
Publisher:	Lippincott Williams & Wilkins
Language:	English
Submitter:	Laura Seidel
Date Deposited:	21 Apr 2014 08:29
Last Modified:	27 Feb 2024 14:29
Publisher DOI:	10.1161/CIRCRESAHA.113.300269
PubMed ID:	23989720
Uncontrolled Keywords:	cardiac, clinical translation, gene therapy, heart failure
URI:	https://boris.unibe.ch/id/eprint/40981