Gene therapy in pediatrics Academic Article uri icon

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abstract

  • There has been a rapid proliferation of gene therapy trials in humans in the last few years. Gene therapy in pediatric patients has included treatment of classical genetic diseases as well as cancer and AIDS. Despite hundreds of trials involving thousands of patients, to date no disease has been cured with gene therapy. Setbacks in these trials have led to the development of new vectors for gene transfer, more specific targeting of therapy and increased efforts to understand the pathophysiology of diseases.

publication date

  • 1998