Which diseases can be treated with gene therapy?
Gene therapy is primarily used in genetic disorders caused by single-gene mutations. Conditions such as Spinal Muscular Atrophy (SMA), hemophilia, beta thalassemia, sickle cell disease, Duchenne muscular dystrophy, and inherited retinal disorders are among the best-known examples. Advanced approaches like CAR-T cell therapies are also being used in cancer treatment. In addition, extensive clinical research is ongoing for HIV, diabetes, and neurological diseases.