UCL Discovery

Abstract

Cardiovascular disease (CVD) is the leading cause of death worldwide; possible treatments include lifestyle modification, risk factor control and statin-mediated reduction of low-density lipoproteins (LDL), the major carrier of plasma cholesterol. Nonetheless, about two-thirds of adverse cardiovascular events continue despite these interventions. Epidemiological data have shown an inverse relationship between cardiovascular event rates and levels of high-density lipoproteins (HDL). This, together with laboratory findings and results of early clinical trials, has led to a shift towards HDL as a therapeutic target for decreasing CVD risk [1,2]. Here, we review recent progress in gene therapy strategies and their practical application to HDL and apolipoprotein (apo) A-I, its major protein constituent. In particular, we focus on the development of adeno-associated virus (AAV) vectors and oligonucleotide-mediated gene editing.