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.
Read the full story here: UK scientists develop brain disease therapy