3-year-old boy battles Hunter Syndrome Received the world’s first stem cell gene therapy treatment
Hunter syndrome It is a rare genetic disorder. which damages the body and brain, on average it affects children between the ages of 10 and 20 years.
This treatment was developed over 10 years at the University of Manchester. and tested at the Royal Manchester Children’s Hospital (RMCH). The boy was treated with gene therapy in February 2025. Over the months following treatment he continued to recover.
Experts use single-dose gene therapy By using children’s stem cells to correct abnormal genes in the laboratory. The modified cells are then injected back into the patient. These modified stem cells are able to produce the missing enzyme.
To treat the brain Breaks down accumulated toxic sugars And hope that in the future it will help prevent dementia and make it 100% curable.
Currently, stem cell treatment plans are another option. Instead expensive enzyme replacement therapy (£375,000 per year) must be done once a week for life.
Source: interestingengineering
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