Lentiviral Vectors--the Promise of Gene Therapy Within Reach?

Abstract

The goal of gene therapy is to modify the genetic material of cells by introducing a therapeutic gene to treat or prevent disease. However, gene therapy has been hampered by the lack of suitable delivery systems (vectors). Existing vectors (all based on viruses) have major problems including poor transduction efficiency, inability to infect nondividing cells, short-term expression of the therapeutic gene and induction of a host immune response. In a Perspective, Amado and Chen discuss the new generation of vectors that are based on lentiviruses such as HIV. They discuss the advantages of such lentiviral vectors but also raise the safety concerns inherent in using such vectors and how these can be overcome.