title: Gene therapy and inherited dyslipidemia.
abstract: 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.
authors.affiliation: Gladstone Institute of Cardiovascular Disease, San Francisco, California, USA.
authors.forename: T
authors.initials: T
authors.lastname: O'Brien