Gene therapy for lipoprotein disorders.
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.
Broedl
Uli C
UC
University of Munich, Department of InternalMedicine II, Marchioninistr. 15, 81377 Munich, Germany. uli.broedl@med.uni-muenchen.de
Rader
Daniel J
DJ
D000818:Animals; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D006949:Hyperlipidemias / Q000235:genetics / Q000628:therapy*; D051379:Mice