Researchers at the University of Manchester developed a promising new approach that could help children with Hunter syndrome, a rare genetic disorder.
More on Hunters syndrome
A missing or malfunctioning IDS enzyme triggers Hunter syndrome.
Without IDS, the body cannot break down complex sugars. As these sugars build up, they cause permanent, progressive damage to the body and brain. Many children develop severe cognitive disabilities. There is no cure, and current treatments are limited and expensive.
New research
Researchers at the University of Manchester collected blood stem cells from bone marrow and genetically corrected the mutation. They also added a tag to the IDS enzyme to help it cross the blood‑brain barrier and deliver therapy directly to the brain.
Brian Bigger, the lead researcher, said the combination therapy corrected bone, joint, and brain disease in the mice.
“We expected the stem cell gene therapy approach to deliver IDS enzyme to the brain,” Bigger said. “How much better the tag made the therapy in the brain surprised us.”
The tag improved enzyme uptake across the blood‑brain barrier. It also increased enzyme uptake into cells and made the enzyme more stable in the bloodstream. Together, these improvements outperformed current technology.
Joshua Davies created a video showing the impact Hunter syndrome called Living Beyond Hope.
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