[HTML][HTML] Anatomical changes and pathophysiology of the brain in mucopolysaccharidosis disorders

BW Bigger, DJ Begley, D Virgintino… - Molecular genetics and …, 2018 - Elsevier
Mucopolysaccharidosis (MPS) disorders are caused by deficiencies in lysosomal enzymes,
leading to impaired glycosaminoglycan (GAG) degradation. The resulting GAG …

Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms

HFE Gleitz, AY Liao, JR Cook, SF Rowlston… - EMBO molecular …, 2018 - embopress.org
The pediatric lysosomal storage disorder mucopolysaccharidosis type II is caused by
mutations in IDS, resulting in accumulation of heparan and dermatan sulfate, causing severe …

Effectiveness of early hematopoietic stem cell transplantation in preventing neurocognitive decline in mucopolysaccharidosis type II: a case series

A Selvanathan, C Ellaway, C Wilson, P Owens… - JIMD Reports, Volume …, 2018 - Springer
The early progressive form of the X-linked disorder, Hunter syndrome or
mucopolysaccharidosis type II (MPS II)(OMIM# 309900), is characterized by cognitive …

Aversive and non-aversive memory impairment in the mucopolysaccharidosis II mouse model

AS Azambuja, L Correa, BP Gabiatti, GR Martins… - Metabolic Brain …, 2018 - Springer
Abstract Hunter syndrome (MPS II, OMIM 309900) is a lysosomal storage disorder due to
deficient iduronate sulphatase activity. Patients present multiple cognitive alterations, and …

[PDF][PDF] DIAGNOSIS METHOD FOR MUCOPOLYSACCHARIDOSES

H Peracha - 2018 - udspace.udel.edu
Mucopolysaccharidoses (MPS) are a group of lysosomal storage diseases caused by the
deficiency of lysosomal enzyme that is required to degrade various glycosaminoglycans …