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DEPHOSPHORYLATED LYSOSOMAL STORAGE DISEASE PROTEINS AND METHODS OF USE THEREOF
专利权人:
发明人:
申请号:
EP13747600.8
公开号:
EP2880156B1
申请日:
2013.07.31
申请国别(地区):
EP
年份:
2017
代理人:
摘要:
Provided are substantially dephosphorylated forms of lysosomal storage disease (LSD) proteins, including dephosphorylated forms of iduronate-2-sulfatase (IDS, or I2D) and iduronidase (IDU), having increased ability to traverse or penetrate the blood brain barrier (BBB) relative to phosphorylated forms of the protein, and p97 conjugates thereof. Also provided are compositions comprising such dephosphorylated LSD proteins and p97 conjugates, and methods of use thereof, for instance, to treat any one or more lysosomal storage diseases, such as Hunter Syndrome (or MPS Type II).
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