UK scientists have developed a new stem cell therapy that could, in the best case scenario, offer a “largely curative” treatment for the deadly genetic brain disease Sanfilippo, as well as a new platform for other neurodegenerative conditions such as Alzheimer’s disease.
A team at Manchester University have used a gene therapy vector to treat Sanfilippo in mice for the first time, and hope to be testing the treatment in humans within two years.
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