Scientists continue to work on new ways to treat thalassemias. The leading research areas currently focus on gene therapy and stem cell transplantation. Stem cells are immature cells that have not yet specialized. Immature stem cells in the bone marrow give rise to red blood cells. Technically, a bone marrow transplant, which is the only cure for thalassemia, is a form of stem cell transplantation. Additionally, researchers are looking at using umbilical cord blood as a source of stem cells. Unlike bone marrow stem cells, umbilical stem cells need not provide a perfect genetic match, meaning there is lower chance of rejection.

Whereas stem cell approaches attempt to replace the defective red blood-cell producing bone marrow, gene therapy techniques are used in an attempt to repair the patient's defective cells. In this case, a normal genetic code, such as for beta protein, is inserted into a patient's stem cell, which would allow that stem cell to produce normal blood cells. There are, however, many obstacles to overcome before this line of research results in clinical therapies. For one, working with stem cells in a laboratory setting is difficult, and stem cell research has been placed under federal restrictions because of concerns over embryonic stem cells. Also, it is difficult to actually get a stem cell to incorporate new genetic material because of the nature of stem cells themselves.

Experts also are examining methods of increasing the production of fetal hemoglobin in patients. Fetal hemoglobin is the type of hemoglobin produced in the body before birth. After a child is born, the body normally changes to making adult hemoglobin. However, a genetic glitch causes some children continue to produce fetal hemoglobin after birth. This has the unintended benefit of making thalassemia less severe in patients who have this condition.