Strategies for targeted gene therapy.

Genetic intervention for the therapy of human disease has long been a dream for scientists and clinicians alike, and the first steps towards reality have already been taken in clinical trials involving over 1000 patients around the world. The technology used in these initial experiments has limited potential for therapeutic effect and it is now appreciated that improvements in targeting gene delivery and gene expression are both required before real clinical benefit is achieved. The enormous advances made in the fields of molecular genetics and molecular biology of the last few years have set the scene for their translation into novel approaches to gene transfer and control, for gene therapy applications.