Enzyme replacement therapy in newborn mucopolysaccharidosis IVA mice: early treatment rescues bone lesions?

We treated mucopolysaccharidosis IVA (MPS IVA) mice to assess the effects of long-term enzyme replacement therapy (ERT) initiated at birth, since adult mice treated by ERT showed little improvement in bone pathology (1). To conduct ERT in newborn mice, we used recombinant human N-acetylgalactosamine...

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Dades bibliogràfiques
Publicat a:Mol Genet Metab
Autors principals: Tomatsu, Shunji, Montaño, Adriana M., Oikawa, Hirotaka, Dung, Vu Chi, Hashimoto, Amiko, Oguma, Toshihiro, Gutiérrez, Monica L., Takahashi, Tatsuo, Shimada, Tsutomu, Orii, Tadao, Sly, William S.
Format: Artigo
Idioma:Inglês
Publicat: 2014
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4256128/
https://ncbi.nlm.nih.gov/pubmed/24953405
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2014.05.013
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