More and more is being said about the incredible possibilities of gene therapy. This relatively young technique was used by Parisian doctors in a boy with congenital sickle cell anemia. The magazine «New England Journal of Medicine» informed about the success of specialists.
The procedure was performed 15 months ago in a 13-year-old boy with sickle cell anemia. Due to the disease, his spleen was removed and both hip joints were replaced with artificial ones. He had to undergo blood transfusions every month.
Sickle cell anemia is a genetic disease. The defective gene changes the shape of the red blood cells (red blood cells) from round to sickle, which causes them to stick together and circulate in the blood, causing damage to internal organs and tissues and causing them to become oxygenated. This results in pain and premature death, and life-saving frequent blood transfusions are also necessary.
Hôpital Necker Enfants Malades in Paris removed the boy’s genetic defect, first by completely destroying his bone marrow, where they are produced. Then they recreated it from the boy’s stem cells, but previously genetically modified them in the laboratory. This procedure involved introducing the correct gene into them with the help of a virus. The bone marrow has regenerated to produce normal red blood cells.
The head of the research, prof. Philippe Leboulch told BBC News that the boy, now almost 15, is doing well and is showing no symptoms of sickle cell anemia. He does not feel any symptoms and does not require hospitalization. But there is no question of full recovery yet. The effectiveness of the therapy will be confirmed by further research and tests on other patients.
Dr. Deborah Gill from the University of Oxford is convinced that the procedure performed by French specialists is a great achievement and a chance for effective treatment of sickle cell anemia.