Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II

BACKGROUND: Mucopolysaccharidosis type II (MPS II, Hunter syndrome), is caused by a deficiency of iduronate-2-sulfatase (IDS). Despite the therapeutic effect of intravenous enzyme replacement therapy (ERT), the central nervous system (CNS) defects persist because the enzyme cannot cross the blood-br...

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Vydáno v:Orphanet J Rare Dis
Hlavní autoři: Cho, Sung Yoon, Lee, Jeehun, Ko, Ah-Ra, Kwak, Min Jung, Kim, Sujin, Sohn, Young Bae, Park, Sung Won, Jin, Dong-Kyu
Médium: Artigo
Jazyk:Inglês
Vydáno: BioMed Central 2015
Témata:
Research
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4628320/
https://ncbi.nlm.nih.gov/pubmed/26520066
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0356-0
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