Tests and Procedures
Gene therapy
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.