Cardiac troponin T promoters lead the way: optimizing cardiac specificity in AAV-mediated gene therapy

Cardiac gene therapy is emerging as a transformative strategy for life-threatening cardiovascular disorders that are refractory to conventional therapies. Genetically mediated diseases such as Danon Disease, hypertrophic cardiomyopathy (HCM), and arrhythmogenic right ventricular cardiomyopathy (ARVC) confer a markedly increased risk for severe arrhythmias, sudden cardiac death, heart failure, and in advanced cases, necessitate cardiac transplantation. Accordingly, there is an urgent and ongoing pursuit of novel therapeutic modalities, such…