Sixty thousand people are diagnosed each year with beta-thalassaemia, requiring them to get blood transfusions for the rest of their lives.
A new gene is in clinical trials, and a recipient has not needed a transfusion for 21 months after having the gene added into his cells.
The therapy works by changing the genes in a patient's own bone marrow. The altered stem cells will not be rejected by the patient, who will now be able to produce the hemoglobin needed.
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