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MATERIALS AND METHODS FOR TREATING DISORDERS ASSOCIATED WITH SULFATASE ENZYMES
专利权人:
BIOSTRATEGIES LC
发明人:
DAVID N. RADIN
申请号:
US15325661
公开号:
US20170191041A1
申请日:
2015.07.10
申请国别(地区):
US
年份:
2017
代理人:
摘要:
The subject invention concerns materials and methods for treating or preventing disease and conditions associated with various sulfatase enzymes that are defective or that are not properly expressed in a person or animal. In one embodiment, the disease is Sanfilippo A (MPS-IIIA) disease. The subject invention also concerns materials and methods for treating or preventing multiple sulfatase deficiency (MSD) in a person or animal. Compounds of the invention include a fusion protein comprising i) a mammalian sulfatase, or an enzymatically active fragment or variant thereof, and ii) a plant lectin or a binding subunit thereof. In a specific embodiment, the mammalian sulfatase is a human sulfatase, or an enzymatically active fragment or variant thereof. Polynucleotides encoding the fusion proteins are also contemplated for the subject invention. The subject invention also concerns materials and methods for producing proteins of the invention.
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