Molecular biology and genetics in cardiovascular research: highlights of 2002

In the future treatment of haemophilia B, a real breakthrough may be a strategy that uses site-specific genomic integration of a gene therapy vector to produce therapeutic levels of human clotting factor IX (FIX). A clinically relevant expression of plasma levels of FIX was noted for over 12 months. The strategy will be applicable for a broad range of therapeutic genes and tissues. Following the concept that angiogenic growth factors could stimulate revascularisation, a highly interesting novel approach to the 'bio-bypass' has been presented that appears to have some unexpected advantages. It was demonstrated that specifically designed transcription factors can regulate gene expression in vivo. Another important finding was that myocardial stress signals all appear to converge to a common downstream target, the class II histone deacetylases. In mice, hypertrophic stimuli proved to lead to the activation of a novel and so far unique cardiac HDAC kinase that phosphorylates the signal-responsive sites in class II HDACs. A major implication is that the cardiomyocytic HDAC kinase could well be a novel therapeutic target for the treatment of hypertrophy and heart failure. And finally, Catherine Verfaillie and her group published a landmark paper demonstrating that pluripotent stem cells that have the potency to differentiate into most, if not all, somatic tissues can also be isolated from adult bone marrow.