The prospect of gene therapy for lipid disorders

The refractory hyperlipoproteinemia that is associated with atherosclerotic vascular disease is a good candidate for somatic gene therapy. The molecular etiology and pathophysiology of these diseases are mostly well understood, the animal models for these diseases have been extensively developed, and the preclinical proof-of-principle gene transfer studies have already been performed. The clinical gene therapy trials performed in the last decade raised the issue of the necessity for the development of refined vectors that can stand for clinical usage. Recent progress in basic researches implies the feasibleness of the development of vectors that provide long-term and highly-efficient gene expression, which would potentially lead to the beginning of a new era in the field of gene therapy for lipid disorders.