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Manchester Research Group on Brink of Developing Treatments

A research group at Manchester University is at the point of developing a range of treatments for childhood genetic diseases having established proof of principle for a number of basic mechanistic approaches. The Manchester based Stem Cell & Neurotherapies group is developing therapies for childhood brain diseases including Hurler and Sanfilippo diseases.

The lab was set up at the Royal Manchester Children’s Hospital and University of Manchester by Dr Rob Wynn,  the late Prof Ed Wraith and Dr Brian Bigger. The nine-strong research team is using a bench to bedside approach to study basic disease mechanisms and translate resulting therapies into treatment for kids with these diseases.

The group has recently described two novel treatments for Sanfilippo disease in work published in Public Library of Science One, and Molecular Therapy. Dr Bigger described how “daily use of the drug genistein is able to delay disease onset and progression in mice with Sanfilippo type B, whilst gene therapy to replace the missing enzyme in stem cells can correct disease in mice with Sanfilippo type A.

Dr Wynn commented “Currently we can use bone marrow transplant as a partly effective treatment for Hurler syndrome but this treatment doesn’t work at all in Sanfilippo disease. By using gene therapy to deliver more of the missing enzyme in a bone marrow transplant we hope that we can develop this approach in a clinical trial for Sanfilippo in the near future and for other similar diseases later on.”

The team is seeking funding for these two clinical trials and further work to continue studying how Hurler and Sanfilippo diseases occur so they can develop more effective treatments in the future. More information can be found at: www.medicine.manchester.ac.uk/staff/BrianBigger