New Stem Cell Gene Therapy Gives Hope to Prevent Inherited Neurological Disease

Source: University of Manchester
Date: 25 July 2013

Summary:

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.