Methods to improve cardiac gene therapy expression

Abstract

Gene therapy strategies are becoming a valuable approach for the treatment of heart failure. Some trials are ongoing and others are being organized. Vascular access in clinical experimentation is still the chosen modality of delivery, but many other approaches are in research and development. A successful gene therapy strategy involves not only the choice of the right vector and gene, but also the correct delivery strategy that allows for transduction of the highest percentage of cardiomyocytes, limited spilling of virus into other organs and the possibility to correlate the amount of injected virus to the rate of the expression within the cardiac tissue. The authors will first concentrate on clarifying what the barriers are that the virus has to overcome in order to reach the nuclei of the target organs and methodologies that have been tested to improve the range of expression.

Myocardial transcription factors in diastolic dysfunction: clues for model systems and disease

“…1b) or altering their DNA-binding activity (reviewed in [25]). In this context, a number of delivery platforms have been described that are translatable to the clinical setting (reviewed in [119]). However, it is necessary to develop more efficient delivery systems for cardioselective (not systemic) targeting of TFs in the diastolic heart.…”