Recombinant human alpha-N-acetylglucosamine-6-sulfatase delivered to Sanfilippo D mice with repeated intracerebroventricular injections corrects CNS pathology
PLoS One. 2025 Jul 25;20(7):e0328941. doi: 10.1371/journal.pone.0328941. eCollection 2025. ABSTRACT Mucopolysaccharidosis type IIID (MPS IIID; Sanfilippo D) is caused by biallelic pathogenic variants in N-acetylglucosamine-6-sulfatase …