MPS IIIA: gene therapy for brain and body

Less than a year after reporting preclinical proof of concept for a systemically delivered mucopolysaccharidosis IIIA gene therapy, researchers at the Autonomous University of Barcelona have shown that intracerebral delivery can substantially improve efficacy in the CNS and treat symptoms in peripheral organs.¹ Esteve S.A., who partially funded the work, is now raising funds to test the approach in the clinic.

Mucopolysaccharidosis IIIA (MPS IIIA)-also called Sanfilippo type A syndrome-is caused by a mutation in N-sulfoglucosamine sulfohydrolase (SGSH) that impairs the ability of SGSH to degrade heparan sulfate glycosaminoglycans (HSGAGs)...