Genetics is playing an increasingly important role in medicine. It has become clear that genetic factors are involved in virtually every disease that afflicts humanity. Although conventional pharmacologic therapy is effective for many diseases, the underlying genetic predispositions that lead to the diseases are not influenced by these therapies. In particular, with few exceptions, therapy is woefully inadequate for diseases that follow a simple mendelian pattern of inheritance and thus are caused primarily by a single genetic defect.
The ultimate goal of treatment of genetic disease is repair of the genetic defect, ie, gene therapy.1,2 In the past few years, there has been an explosion of interest in this new form of medical treatment. As gene therapy increasingly moves from research laboratories to clinical trials, it will inevitably become standard therapy for many diseases commonly seen by general pediatricians, such as cystic fibrosis (CF), hemophilia, and sickle cell anemia. Thus,