my niece was adopted from china and has a disease called thalassemia, my brother just told me the other day starting in may she will undergo gene therapy. She requires 18 blood transfusions a year, She is seven. the blood transfusions are filling her body with iron and her organs are shutting down, the only way forward is a new gene therapy treatment or a bone marrow transplant. The gene therapy has a lower risk of being fatal but requires an intense chemo therapy. Please keep my brothers family in your prayers moving forward.