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Article

Enzyme replacement therapy (ERT) procedure for mucopolysaccharidosis type II (MPS II) by intraventricular administration (IVA) in murine MPS II.

Molecular genetics and metabolism - 1 Sept 2012

Higuchi Takashi, Shimizu Hiromi, Fukuda Takahiro, Kawagoe Shiho, Matsumoto Juri, Shimada Yohta, Kobayashi Hiroshi, Ida Hiroyuki, Ohashi Toya, Morimoto Hideto, Hirato Tohru, Nishino Katsuya, Eto Yoshikatsu

Abstract excerpt

Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is a lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase (IDS) and is characterized by the accumulation of glycosaminoglycans (GAGs). MPS II has been treated by hematopoietic stem cell therapy (HSCT)/enzyme replacement therapy (ERT), but its effectiveness in the central nervous system (CNS) is limited because of poor enzyme uptake...

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Identifiers

PMID: 22704483
DOI: 10.1016/j.ymgme.2012.05.005

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IDS

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