Enzyme Replacement in a Human Model of Mucopolysaccharidosis IVA In Vitro and Its Biodistribution in the Cartilage of Wild Type Mice

Open Access

Peer-reviewed

Research Article

Melita Dvorak-Ewell,

Affiliation BioMarin Pharmaceutical Inc., Novato, California, United States of America
⨯
Dan Wendt,

Affiliation BioMarin Pharmaceutical Inc., Novato, California, United States of America
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Chuck Hague,

Affiliation BioMarin Pharmaceutical Inc., Novato, California, United States of America
⨯
Terri Christianson,

Affiliation BioMarin Pharmaceutical Inc., Novato, California, United States of America
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Vish Koppaka,

Affiliation BioMarin Pharmaceutical Inc., Novato, California, United States of America
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Danielle Crippen,

Affiliation Buck Institute, Novato, California, United States of America
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Emil Kakkis,

Affiliation BioMarin Pharmaceutical Inc., Novato, California, United States of America
⨯
Michel Vellard

* E-mail: mvellard@bmrn.com

Affiliation BioMarin Pharmaceutical Inc., Novato, California, United States of America
⨯

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Chondrocytes
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Collagens
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Lysosomes
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Cartilage
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Growth plate
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Articular cartilage
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Gene expression
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Heart
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