The maturation of human gene therapy

Human gene therapy is two things. It is the concept that human disease might be treated at the level of underlying genetic targets rather than at the level of aberrant metabolism, and it is the implementation of that concept toward a clinical reality. The conceptual aspect is established gene therapy has become an accepted central driving force in medicine. The second aspect—that of converting the concepts into practical tools for human gene therapy—is maturing rapidly. Over the past several years, the level of expectation had risen to unrealistic proportions and recent initial clinical trials produced disappointment. These early clinical results should, however, be viewed not as failures, but rather as deliberate progress along the learning curve in this new and difficult field of biomedical science.