Prof Bigger said “We expected the stem cell gene therapy approach to deliver IDS enzyme to the brain, as we have shown previously for another disease: Sanfilippo types A and B, but we were really surprised to discover how much better the tag made the therapy in the brain.

“It turns out that the tag didn’t only improve enzyme uptake across the blood brain barrier, but also improved uptake of the enzyme into cells and it appeared to be more stable in the bloodstream – all improvements on current technology.”

The team are part of the recently awarded InnovateUK Manchester Advanced Therapy Centre Hub (iMATCH), and aim to develop this therapy within this framework to take it to clinical trial.

A video describing the effects of the disease and potential of gene therapy can be seen here in Living Beyond Hope: A Short Documentary by Joshua Davies.

He added: “We are extremely grateful to our charity funders without whom this work would not have been possible, including the Isaac Foundation, the National MPS Society and the Newlife Foundation.

 



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