Clearance of heparan sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice

Mucopolysaccharidosis II (MPS II), a lysosomal storage disease caused by mutations in iduronate-2-sulfatase (IDS), is characterized by a wide variety of somatic and neurologic symptoms. The currently approved intravenous enzyme replacement therapy with recombinant IDS (idursulfase) is ineffective fo...

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Pubblicato in:Mol Ther
Autori principali: Morimoto, Hideto, Kida, Sachiho, Yoden, Eiji, Kinoshita, Masafumi, Tanaka, Noboru, Yamamoto, Ryuji, Koshimura, Yuri, Takagi, Haruna, Takahashi, Kenichi, Hirato, Tohru, Minami, Kohtaro, Sonoda, Hiroyuki
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2021
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC8116601/
https://ncbi.nlm.nih.gov/pubmed/33508431
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2021.01.027
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