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Blood-Producing Stem Cells Transformed for Beta-Thalassemia Treatment
The groundbreaking treatment for beta-thalassemia, a debilitating genetic disease affecting millions worldwide, has been approved in the UK. This innovative approach harnesses the power of gene editing to restore normal hemoglobin production in patients.
How it works:
- Blood-producing stem cells are harvested from the patient.
- In the laboratory, the genetic switch that controls hemoglobin production is modified using CRISPR technology.
- The old, malfunctioning stem cells are eliminated with chemotherapy.
- The reprogrammed stem cells are then returned to the body.
Potential for a Life-Changing Impact:
- Patients will no longer require regular blood transfusions, significantly improving their quality of life.
- Clinical trials have shown remarkable results, with 49 out of 52 patients remaining transfusion-free for at least a year.
Cost and Availability:
- The treatment is estimated to cost £1.6 million per patient, but the UK’s National Institute for Health and Care Excellence has deemed it cost-effective.
- The therapy is currently available at seven specialized centers and is accessible to patients over 12 years old.
Hope for the Future:
- This revolutionary treatment offers new hope for the estimated 460 beta-thalassemia patients in the UK.
- Discussions are ongoing to explore the potential of this technology for other genetic hemoglobin disorders like sickle cell anemia.
Impact on the Thalassemia Community:
- Romaine Maharaj, executive director of the UK Thalassemia Society, expressed immense joy, calling the treatment a “beacon of hope” for patients.
- This groundbreaking achievement is a testament to the tireless efforts of researchers and healthcare professionals to combat this debilitating disease.