A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II

Mucopolysaccharidosis II (MPS II) is an X-linked recessive lysosomal storage disease caused by mutations in the iduronate-2-sulfatase (IDS) gene. Since IDS catalyzes the degradation of glycosaminoglycans (GAGs), deficiency in this enzyme leads to accumulation of GAGs in most cells in all tissues and...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Sonoda, Hiroyuki, Morimoto, Hideto, Yoden, Eiji, Koshimura, Yuri, Kinoshita, Masafumi, Golovina, Galina, Takagi, Haruna, Yamamoto, Ryuji, Minami, Kohtaro, Mizoguchi, Akira, Tachibana, Katsuhiko, Hirato, Tohru, Takahashi, Kenichi
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5993955/
https://ncbi.nlm.nih.gov/pubmed/29606503
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.02.032
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