Owen, J.S.; (2005) ApoE gene therapy: an overview and update. (Commentaries ). International Atherosclerosis Society

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Abstract

Atherosclerosis remains the leading cause of death in industrialized societies. Apolipoprotein E (ApoE) is an attractive candidate to treat hypercholesterolemia and coronary heart disease, as it is a circulating protein with pleiotropic atheroprotective actions. Here, we describe several "gene addition" approaches and on-going developments to achieve efficient delivery and long-term expression. The use of recombinant viruses is discussed, including adeno-associated viral vectors (AAV) where technological advances now allow the cross-packaging of different AAV serotypes. Nonviral delivery systems are also described, including plasmids and cell-based therapy. Finally, a radical, alternative technology to gene addition, which has the potential for permanent cure in many genetic diseases, is reviewed: "targeted gene repair", which aims to correct underlying point mutations in-situ. Synthetic oligonucleotides are designed to bind specifically to defective DNA, enabling the cell's own mismatch machinery to recognize and repair the faulty DNA. Although such gene editing technology has great potential it remains inconsistent and difficult to reproduce.

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