Transcriptional Targeting Approaches in Cardiac Gene Transfer Using AAV Vectors

Pathogens. 2023 Oct 30;12(11):1301. doi: 10.3390/pathogens12111301.

Abstract

Cardiac-targeted transgene delivery offers new treatment opportunities for cardiovascular diseases, which massively contribute to global mortality. Restricted gene transfer to cardiac tissue might protect extracardiac organs from potential side-effects. This could be mediated by using cis-regulatory elements, including promoters and enhancers that act on the transcriptional level. Here, we discuss examples of tissue-specific promoters for targeted transcription in myocytes, cardiomyocytes, and chamber-specific cardiomyocytes. Some promotors are induced at pathological states, suggesting a potential use as "induction-by-disease switches" in gene therapy. Recent developments have resulted in the identification of novel enhancer-elements that could further pave the way for future refinement of transcriptional targeting, for example, into the cardiac conduction system.

Keywords: adeno-associated virus (AAV) vectors; cardiac enhancers; cardiac promoters; gene therapy; transcriptional targeting.

Publication types

  • Review

Grants and funding

This research received no external funding.