Gene therapy for lipoprotein disorders

Abstract

Existing approaches to the treatment of refractory hypercholesterolaemia, severe hypertriglyceridaemia, low levels of high-density lipoprotein cholesterol and certain inherited disorders of intracellular lipid metabolism are ineffective in a substantial number of patients. Somatic gene therapy is considered to be a potential approach to the therapy of several of these lipid disorders. In many cases preclinical proof-of-principle studies have already been performed, and in one (homozygous familial hypercholesterolaemia) a clinical trial has been conducted. Other clinical gene therapy trials for dyslipidaemia are likely to be initiated within the next several years.