Gene therapy: future or flop

Many pediatric diseases have now reached a therapeutic plateau using standard therapy. Gene therapy has emerged as an exciting new means to achieve specific therapeutic benefit. Although there have been important and promising breakthroughs in recent clinical trials, there have been some serious setbacks that have tempered this initial excitement. In this review, we discuss the important developments in the field of gene therapy as it applies to various pediatric diseases and relate the recent successes and failures to the future potential of gene therapy as a medical therapeutic application.