Results

The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in people have shown some success in treating certain diseases, such as:

  • 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
  • Targeting the correct cells
  • Reducing the risk of side effects

Gene therapy continues to be a very important and active area of research aimed at developing new, effective treatments for a variety of diseases.