| Project Name | The use of Gene Therapy in Treatment of Thalassemia |
| Faculty/Department | Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University |
| Background and Significance | Thalassemia anemia is the most common hereditary disease in the world, causing newborn children to experience complications in development. Such difficulties can be considered a burden for families and society, as treatment and medication for the disease has to be administered throughout one's lifespan. To prevent the negative impacts on the quality of life of each patient with Thalassemia, researchers from Ramathibodi have developed a method of gene therapy where the stem cells of the patient are edited in-vitro, and transplanted back into the patient again. This form of treatment is the first of its kind, in the world, to successfully cure Thalassemia. |
| Study Group | Thalassemia anemia patients, Thai and foreign |
| Purpose | To find new methods of treating Thalassemia, the most common hereditary disease in Thailand. |
| Sponsors | - |
| Related Departments | Doctors and researchers from the University of Paris and Harvard Medical School |
| Related Stakeholders | Healthcare systems worldwide |
| Level of Cooperation | By nation |
| Results for Further Use | Using the development of curing Thalassemia without the use of blood transfusions and iron supplements as a method and model to cure other genetic diseases. |
| Related Links | https://med.mahidol.ac.th/rama_hospital/th/services/knowledge/01202020-1601 |
| Related Sustainable Development Goals (SDGs) | - |
