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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Bibliografske podrobnosti
izdano v:Mol Genet Metab
Main Authors: 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
Jezik:Inglês
Izdano: 2014
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Article
Online dostop: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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