[Comment] Intracerebral gene therapy for mucopolysaccharidosis type IIIB syndrome

The devastating paediatric-onset lysosomal storage disorder mucopolysaccharidosis type III syndrome (also known as Sanfilippo syndrome), has been viewed as untreatable. Rapidly progressing neurological impairments lead to death, typically in the second decade of life. In The Lancet Neurology, Marc Tardieu and colleagues1 report the outcomes of a phase 1/2 clinical trial of intracerebral gene therapy with a recombinant adenoassociated viral vector serotype 2/5 encoding human α-N-acetylglucosaminidase (NAGLU) in four young children (age 20–53 months) with mucopolysaccharidosis type IIIB syndrome.

Source: Lancet Neurology - July 13, 2017 Category: Neurology Authors: Kim Hemsley, Adeline Lau Tags: Comment Source Type: research