Gene therapy trial in Manchester shows promise for curing Hunter's Syndrome

rtl.nl (Dutch)

A 3-year-old boy appears cured of the previously incurable Hunter's Syndrome after a groundbreaking gene therapy trial in Manchester. Researchers used the boy's own stem cells, modified in a lab with a virus carrying the missing gene, to produce the necessary enzyme. Doctors are astonished by his progress. Hunter's Syndrome affects approximately 1 in 100,000 children, typically leading to severe physical and cognitive decline. This treatment offers significant hope for future patients.


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