Enhancement of Drug Delivery: Enzyme-replacement Therapy for Murine Morquio A Syndrome

Mucopolysaccharidosis IVA (MPS IVA, Morquio A disease) is an inherited lysosomal storage disorder that features skeletal chondrodysplasia caused by deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Human GALNS was bioengineered with the N-terminus extended by the hexaglutamate sequenc...

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Bibliographische Detailangaben
Hauptverfasser: Tomatsu, Shunji, Montaño, Adriana M, Dung, Vu Chi, Ohashi, Amiko, Oikawa, Hirotaka, Oguma, Toshihiro, Orii, Tadao, Barrera, Luis, Sly, William S
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2010
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Original Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2889747/
https://ncbi.nlm.nih.gov/pubmed/20332769
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.32
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