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...
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Format: | Patent |
Sprache: | eng ; fre ; ger |
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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. |
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