COMPOSITIONS, DEVICES AND METHODS FOR TREATING MPS VI DISEASE

Described herein are mammalian cells engineered to express and secrete an ARSB protein and optionally a sialytransferase protein, as well as compositions, implantable devices and device preparations comprising the engineered cells, and methods of making and using the same for treating MPS VI disease...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: PEARSON, Erika, MAKINO, Elina
Format: Patent
Sprache:eng ; fre ; ger
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Beschreibung
Zusammenfassung:Described herein are mammalian cells engineered to express and secrete an ARSB protein and optionally a sialytransferase protein, as well as compositions, implantable devices and device preparations comprising the engineered cells, and methods of making and using the same for treating MPS VI disease.