Gene therapy and inherited dyslipidemia.
OBJECTIVE
To explore the current status and future potential of gene therapy for the inherited dyslipidemias.


METHODS
A brief overview of the inherited dyslipidemias, a review of the currently available means of transferring genetic material in vivo, and a discussion of two examples of conditions in which gene therapy may be useful--familial hypercholesterolemia and reduced high-density lipoprotein cholesterol syndromes--are presented.


RESULTS
Although substantial progress has been made in the management of inherited dyslipidemia, optimal treatment regimens are not available in all cases. Gene therapy has recently emerged as a potential solution to some of these problems. For gene therapy to be successful, several factors are necessary: an efficient means of gene transfer, long-term transgene expression, and lack of toxicity. Although the feasibility of this approach has been demonstrated, currently available vectors have a number of technical and safety limitations.


CONCLUSIONS
Gene therapy for inherited dyslipidemias has many technical hurdles that must be overcome before it will have widespread clinical application.
O'Brien
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Gladstone Institute of Cardiovascular Disease, San Francisco, California, USA.