PMID- 26051019
OWN - NLM
STAT- MEDLINE
DCOM- 20161103
LR  - 20161230
IS  - 1096-7206 (Electronic)
IS  - 1096-7192 (Linking)
VI  - 117
IP  - 2
DP  - 2016 Feb
TI  - A method for measuring disease-specific iduronic acid from the non-reducing end 
      of glycosaminoglycan in mucopolysaccharidosis type II mice.
PG  - 140-3
LID - S1096-7192(15)30008-1 [pii]
LID - 10.1016/j.ymgme.2015.05.009 [doi]
AB  - Mucopolysaccharidosis type II (MPS II) is an X-linked lysosomal storage disorder 
      arising from deficiency of iduronate-2-sulfatase (IDS), which results in 
      progressive accumulation of glycosaminoglycans (GAGs) in multiple tissues. 
      Accumulated GAGs are generally measured as the amount of total GAGs. However, we 
      recently demonstrated that GAG accumulation in the brain of MPS II model mice 
      cannot be reliably detected by conventional dye-binding assay measuring total 
      GAGs. Here we developed a novel quantitative method for measurement of 
      disease-specific GAGs based on the analysis of 2-sulfoiduronic acid levels 
      derived from the non-reducing terminal end of the polysaccharides by using 
      recombinant human IDS (rhIDS) and recombinant human iduronidase (rhIDUA). This 
      method was evaluated on GAGs obtained from the liver and brain of MPS II mice. 
      The GAGs were purified from tissue homogenates and then digested with rhIDS and 
      rhIDUA to generate a desulfated iduronic acid from their non-reducing terminal 
      end. HPLC analysis revealed that the generated iduronic acid levels were markedly 
      increased in the liver and cerebrum of the MPS II mice, whereas the uronic acid 
      was not detected in wild-type mice. These results indicate that this assay 
      clearly detects the disease-specific GAGs in tissues from MPS II mice.
CI  - Copyright (c) 2015 Elsevier Inc. All rights reserved.
FAU - Shimada, Yohta
AU  - Shimada Y
AD  - Division of Gene Therapy, Research Center for Medical Sciences, The Jikei 
      University School of Medicine, Tokyo 105-8461, Japan. Electronic address: 
      shimada_y@jikei.ac.jp.
FAU - Wakabayashi, Taichi
AU  - Wakabayashi T
AD  - Division of Gene Therapy, Research Center for Medical Sciences, The Jikei 
      University School of Medicine, Tokyo 105-8461, Japan; Department of Pediatrics, 
      The Jikei University School of Medicine, Tokyo 105-8461, Japan.
FAU - Akiyama, Kazumasa
AU  - Akiyama K
AD  - Division of Gene Therapy, Research Center for Medical Sciences, The Jikei 
      University School of Medicine, Tokyo 105-8461, Japan; Department of Pediatrics, 
      Kitasato University Graduate School of Medicine, Kanagawa, Japan.
FAU - Hoshina, Hiroo
AU  - Hoshina H
AD  - Division of Gene Therapy, Research Center for Medical Sciences, The Jikei 
      University School of Medicine, Tokyo 105-8461, Japan; Department of Pediatrics, 
      The Jikei University School of Medicine, Tokyo 105-8461, Japan.
FAU - Higuchi, Takashi
AU  - Higuchi T
AD  - Division of Gene Therapy, Research Center for Medical Sciences, The Jikei 
      University School of Medicine, Tokyo 105-8461, Japan.
FAU - Kobayashi, Hiroshi
AU  - Kobayashi H
AD  - Division of Gene Therapy, Research Center for Medical Sciences, The Jikei 
      University School of Medicine, Tokyo 105-8461, Japan; Department of Pediatrics, 
      The Jikei University School of Medicine, Tokyo 105-8461, Japan.
FAU - Eto, Yoshikatsu
AU  - Eto Y
AD  - Advanced Clinical Research Center, Institute of Neurological Disorders, Kanagawa, 
      Japan.
FAU - Ida, Hiroyuki
AU  - Ida H
AD  - Division of Gene Therapy, Research Center for Medical Sciences, The Jikei 
      University School of Medicine, Tokyo 105-8461, Japan; Department of Pediatrics, 
      The Jikei University School of Medicine, Tokyo 105-8461, Japan.
FAU - Ohashi, Toya
AU  - Ohashi T
AD  - Division of Gene Therapy, Research Center for Medical Sciences, The Jikei 
      University School of Medicine, Tokyo 105-8461, Japan; Department of Pediatrics, 
      The Jikei University School of Medicine, Tokyo 105-8461, Japan.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20150521
PL  - United States
TA  - Mol Genet Metab
JT  - Molecular genetics and metabolism
JID - 9805456
RN  - 0 (Biomarkers)
RN  - 0 (Glycosaminoglycans)
RN  - 3402-98-0 (Iduronic Acid)
RN  - EC 3.1.6.13 (Iduronate Sulfatase)
RN  - EC 3.2.1.76 (Iduronidase)
SB  - IM
MH  - Animals
MH  - Biomarkers/metabolism
MH  - Cerebrum/metabolism
MH  - Enzyme Replacement Therapy
MH  - Female
MH  - Glycosaminoglycans/*metabolism
MH  - Humans
MH  - Iduronate Sulfatase/chemistry/therapeutic use
MH  - Iduronic Acid/chemistry/*metabolism
MH  - Iduronidase/chemistry/therapeutic use
MH  - Liver/metabolism
MH  - Mice, Inbred C57BL
MH  - Mucopolysaccharidosis II/*diagnosis/drug therapy/metabolism
OTO - NOTNLM
OT  - Glycosaminoglycan
OT  - HPLC
OT  - Iduronic acid
OT  - MPS II
OT  - Mucopolysaccharidosis type II
EDAT- 2015/06/09 06:00
MHDA- 2016/11/04 06:00
CRDT- 2015/06/09 06:00
PHST- 2015/02/12 00:00 [received]
PHST- 2015/05/01 00:00 [revised]
PHST- 2015/05/01 00:00 [accepted]
PHST- 2015/06/09 06:00 [entrez]
PHST- 2015/06/09 06:00 [pubmed]
PHST- 2016/11/04 06:00 [medline]
AID - S1096-7192(15)30008-1 [pii]
AID - 10.1016/j.ymgme.2015.05.009 [doi]
PST - ppublish
SO  - Mol Genet Metab. 2016 Feb;117(2):140-3. doi: 10.1016/j.ymgme.2015.05.009. Epub 
      2015 May 21.