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Research team helps children with MPS

A stem cell research laboratory for mucopolysaccharide (MPS) diseases is investigating treatments which may help around 400 children in Manchester - and many more nationally. The lab was set up at the Royal Manchester Children’s Hospital by Drs Rob Wynn, Ed Wraith and Brian Bigger. The nine-strong research team is using a multidisciplinary approach to investigate stem cell and gene therapies to improve bone marrow transplantation in MPS and related diseases. MPS diseases are genetic disorders which affect 1 in 26,000 people, mainly children. In each type of the disease, the patient lacks a specific enzyme needed to break down glycosaminoglycans (GAGs), types of carbohydrate that are essential in connective tissues and are used by the body to help cells to communicate with each other. Explained Dr Bigger: “These GAGs are essential for the body to work normally, but when too many build up, as happens in cells from patients with MPS diseases, they cause severe progressive mental decline, heart and other problems as well as bone and joint disease in some cases. Severe forms of MPS usually result in death in early childhood.” “Milder forms of MPS can be treated with weekly injections of the missing enzyme. However, most severe forms of MPS are currently untreatable as the enzyme supplied in the blood is not able to cross into the brain or to correct bone defects. The exception is Hurler syndrome (MPS IH) which can be treated with a bone marrow transplant. Our team is looking at ways to reduce the risks and side effects involved in bone marrow transplants and to increase success rates. We’re also trying to develop gene therapies for untreatable forms of MPS.” Funded by the UK MPS Society and other national MPS and children’s charities, the research group is based in dedicated laboratory facilities at the Royal Manchester Children’s Hospital.