Levels of glycosaminoglycans in the cerebrospinal fluid of healthy young adults, surrogate-normal children, and Hunter syndrome patients with and without cognitive impairment

Christian J. Hendriksz*, Joseph Muenzer, Adeline Vanderver, Jonathan M. Davis, Barbara K. Burton, Nancy J. Mendelsohn, Nan Wang, Luying Pan, Arian Pano, Ann J. Barbier

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

17 Scopus citations

Abstract

In mucopolysaccharidoses (MPS), glycosaminoglycans (GAG) accumulate in tissues. In MPS II, approximately two-thirds of patients are cognitively impaired. We investigated levels of GAG in cerebrospinal fluid (CSF) in different populations from four clinical studies (including NCT00920647 and NCT01449240). Data indicate that MPS II patients with cognitive impairment have elevated levels of CSF GAG, whereas those with the attenuated phenotype typically have levels falling between those of the cognitively affected patients and healthy controls.

Original languageEnglish (US)
Pages (from-to)103-106
Number of pages4
JournalMolecular Genetics and Metabolism Reports
Volume5
DOIs
StatePublished - Dec 1 2015

Keywords

  • Cerebrospinal fluid
  • Cognitive impairment
  • Glycosaminoglycans
  • Idursulfase
  • Inherited metabolic disease
  • Lysosomal storage disease
  • Mucopolysaccharidosis II

ASJC Scopus subject areas

  • Molecular Biology
  • Genetics
  • Endocrinology

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