International
A gene therapy treatment for kids with a severe immune disorder has shown promise in clinical trials in the US and UK, with 100 per cent of kids surviving two and three years after the treatment. Kids with the disorder, known as severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), can die from infections by the age of two if they don't receive treatment. Treatment often involves stem cell transplants from a related donor, but such donors are available for only approximately 20 per cent of patients. This new study used the patient's own stem cells, which were then genetically modified to correct the deficiency before they were put back into the patients. The study found that after the gene therapy treatment, 90 per cent of the patients in the U.S. studies and 100 per cent of those in the U.K. study were also able to stop their immune system replacement therapy.
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