BOIVIN, DOMINIQUE,CASTAIGNE, JEAN-PAUL,DEMEULE, MICHEL
申请号：
EP13803602
公开号：
EP2861729A4
申请日:
2013.06.14
申请国别(地区):
EP
年份:
2016
代理人:
摘要：
The present invention is related to a compound that includes (a) &alpha；-L-iduronidase (IDUA), fragment, or analog thereof and (b) a targeting moiety, for example, where compound is a fusion protein including IDUA and Angiopep-2. In certain embodiments, these compounds, owning to the presence of the targeting moiety can crossing the blood-brain barrier or accumulate in the lysosome more effectively than the enzyme alone. The invention also features methods for treating mucopolysaccharidosis type I (MPS-I) using such compounds.
