Please help. My daughter receives blood transfusions to live. Gene therapy can cure this. Please help us !!
Beta thalassemia major (Cooley’s anemia)--both (two) beta chain genes have deletions, causing the most severe type of beta thalassemia. Thalassemia major patients need frequent blood transfusions and may not survive a normal lifespan. During the first one to two years of life, they can be pale, fussy, have a poor appetite, and have many infections. Without treatment, the spleen, liver, and heart become enlarged, and bones can become thin, brittle, and deformed. A major problem is the build up of iron in the heart and other organs, resulting in heart failure for some patients in their teens or early 20s.