Stem cell gene therapy was used to treat a fatal genetic brain disease in mice
25 Jul 2013 Scientists from The University of Manchester have used stem cell gene therapy to treat a fatal genetic brain disease in mice for the first time. The method was used to treat Sanfilippo - a fatal inherited condition which causes progressive dementia in children - but could also benefit several neurological, genetic diseases. Researchers behind the study, published in the journal Molecular Therapy this month, are now hoping to bring a treatment to trial in patients within two years. Sanfilippo, a currently untreatable mucopolysaccharide (MPS) disease, affects one in 89,000 children in the United Kingdom, with sufferers usually dying in their mid-twenties. It is caused by the lack of SGSH enzyme in the body which helps to breakdown and recycle long chain sugars, such as heparan sulphate (HS). Children with the condition build up and store excess HS throughout their body from birth which affects their brain and results in progressive dementia and hyperactivity, followed by losing the ability to walk and swallow. Dr Brian Bigger, from the University of Manchester's Institute of Human Development who led the research, said bone marrow transplants had been used to correct similar HS storage diseases, such as Hurler syndrome, by transplanting normal cells with the missing enzyme but the technique did not work with Sanfilippo disease.
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