Article type:
News
Estimated read time:
1 min min read
Published:
22/09/2025
Recent advances in in-utero blood transfusion technology have made it possible for babies diagnosed with alpha thalassemia major—a severe, inherited blood disorder—to survive birth. However, these children require lifelong specialized care due to their inability to produce hemoglobin, leading to serious health issues and dependence on blood transfusions.
A UCLA research team, led by Dr. Donald Kohn, is developing a potentially curative, one-time stem cell gene therapy for alpha thalassemia major. This therapy involves collecting the patient’s own blood stem cells, inserting the missing alpha-globin gene, and returning the corrected cells to the patient. This approach aims to restore normal hemoglobin production and eliminate the need for donor transplants, which are difficult to match and carry significant risks.
The therapy has shown promising results in preclinical studies, successfully restoring healthy hemoglobin levels in patient cells and offering hope for a lifelong cure.
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