7.2.1 Gene therapy for the treatment of diseases
Gene therapy involves the delivery of healthy genes into an infected patient's cells as a drug to treat the disease.
Research on gene therapy is currently used for diseases caused by single genes, e.g. cystic fibrosis, haemophilia, muscular dystrophy, thalassemia and sickle cell anaemia.
Problems associated with gene therapy include its short-lived response therefore requiring multiple treatments and the possibility of inducing an immune response by the patient.