Beta Thalassemia Gene Therapy
Individuals with beta thalassemia major, ages 18 to 35, who are receiving regular transfusion therapy may be able to participate.
Individuals with beta thalassemia major, ages 18 to 35, who are receiving regular transfusion therapy may be able to participate.
Patients ages 10 years and older with thalassemia and low risk myelodysplastic syndrome who receive regular red cell transfusions and have associated iron overload are eligible to participate.
Subjects ages 18 years and older with beta-thalassemia requiring chronic transfusions with associated iron overload requiring chelation therapy are eligible to participate.