Blood Home > Gene Therapy for Thalassemia Major

Research scientists studying thalassemia are currently working on various treatment options, including gene therapy for thalassemia major. The hope is that one may eventually lead to a cure for the disease. Two possibilities of gene therapy for thalassemia major include inserting a normal beta-globin gene into stem cells and using medications to encourage fetal hemoglobin production.

An Introduction to Gene Therapy for Thalassemia Major

Thalassemia research scientists are working to develop a gene therapy that may offer a cure for thalassemia. They are currently looking at two gene therapy treatments:
 
  • Beta-globin gene and stem cells
  • Medications and fetal hemoglobin.
 

Gene Therapy for Thalassemia Major: Beta-Globin and Stem Cells

This type of gene therapy for thalassemia major might involve inserting a normal beta-globin gene (the gene that is abnormal in this disease) into the patient's stem cells, which are the immature bone marrow cells that are the precursors of all other cells in the blood.
 

Gene Therapy for Thalassemia Major: Medications and Fetal Hemoglobin

Another form of gene therapy for thalassemia major could involve using drugs or other methods to reactivate the patient's genes that produce fetal hemoglobin, which is the form of hemoglobin found in fetuses and newborns.
 
Scientists hope that spurring production of fetal hemoglobin will compensate for the patient's deficiency of adult hemoglobin.
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Last reviewed by: Arthur Schoenstadt, MD
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