Gene therapy for disorders affecting children, progress and potential.

For over two decades gene therapy has been actively pursued as a treatment modality for the inherited diseases that affect the paediatric population, however, it is still to make a real impact in the clinic. There are many reasons for this including inadequate technology and a lack of understanding of the biological complexities that impact on the efficiency of gene delivery and its outcomes, both positive and negative. However, recent progress is now addressing these issues and indicates that these problems can be overcome, and that gene therapy will play a significant role in the treatment of at least some of these disorders. This review will first give a short overview of relevant gene delivery technologies, what strategies can be used and which diseases are potential targets for gene therapy, and then illustrate several specific diseases for which gene therapy is actively being developed.