Gene-Therapy Delivery Strategies in Cardiology

Abstract

Clinical gene-therapy approaches in cardiology have not fulfilled their promise in randomized, controlled trials, so far, despite striking effects in preclinical models. Lack of clinical success appears not to be related to an unexpected low potency of the therapeutic factors itself in humans, but has rather been attributed to limitations of the vector systems used to transfer the DNA, as well as application modes of the vector itself. Therefore, novel delivery strategies are required with increased efficiency and increased specificity. Recent improvements of vectors using targeting approaches in addition to the development of novel application strategies for cardiac or vascular gene transfer will improve gene delivery in future clinical approaches.

Keywords: :

• adeno-associated virus
• adenovirus
• animal models
• clinical trials
• endothelium
• gene transfer
• heart
• nonviral vector

Financial & competing interests disclosure

OJ Müller is funded by the Deutsche Forschungsgemeinschaft (Mu 1654/3–2) and Bundesministerium für Bildung und Forschung (01GU0527). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.