Teen's life transformed by gene editing
Summary
A new gene editing treatment has helped teenagers with blood disorders like thalassemia and sickle cell disease by changing their stem cells to produce more healthy hemoglobin. This treatment reduces or removes the need for frequent blood transfusions and improves their quality of life.Key Facts
- Vian, 16, has thalassemia and no longer needs monthly blood transfusions after gene editing treatment.
- The treatment edits patients’ own stem cells to produce more fetal (baby) hemoglobin, which carries oxygen in the blood.
- The edited stem cells are given back to patients after chemotherapy.
- The treatment is described as a "functional cure," meaning it controls symptoms but does not completely eliminate the disease.
- The treatment is available at three children’s hospitals in the country.
- JoelSamuel, 14, with sickle cell disease, is starting the same gene editing process and hopes it will reduce his pain and improve his energy.
- The gene editing technique offers young patients the chance to live more normal and active lives.
- Doctors see this treatment as a major medical advancement after decades of research.
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