The possibilities of gene therapy hold much promise. To date, however, that promise has not been realized.

Clinical trials of gene therapy in people have shown some success in treating:

  • Severe combined immune deficiency
  • Hemophilia
  • Blindness caused by retinitis pigmentosa
  • Leukemia

But, several significant barriers stand in the way of gene therapy becoming a reliable form of treatment, including:

  • Finding a reliable way to get genetic material into cells
  • Reducing the risk of side effects
  • Targeting the correct cells

Gene therapy continues to be a very active area of research.

Jan. 05, 2013