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A UK toddler with a rare genetic condition was the first person to receive a new gene therapy that appears to halt disease progression.
Oliver, now three years old, has Hunter syndrome, an inherited genetic disorder that leads to physical and cognitive decline. A faulty gene leads to reduced production of an important enzyme that breaks down complex sugar molecules. Over time, these build up throughout the body, causing physical pain, cognitive decline and reduced life expectancy, typically 10-20 years.
'For many years, we have performed bone marrow transplants for children with Hunter Syndrome and similar illnesses. However, these are difficult procedures that can only deliver as much enzyme as the donor's blood naturally has,' said Professor Rob Wynn, consultant paediatric haematologist and director of the paediatric bone marrow transplant programme at Manchester University NHS Foundation Trust. 'Gene therapy is not only safer and more effective, but it enables us to use the child's own cells, which cuts out the need to find a donor, and means we can produce more enzyme for the patient,' he explained.
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