发明名称 MATERIALS AND METHODS FOR TREATING DISORDERS ASSOCIATED WITH SULFATASE ENZYMES
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 -III A) 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.
申请公布号 WO2016007909(A3) 申请公布日期 2016.03.24
申请号 WO2015US40030 申请日期 2015.07.10
申请人 BIOSTRATEGIES LC 发明人 RADIN, DAVID, N.
分类号 A61K38/16 主分类号 A61K38/16
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