Boivin, Dominique,Castaigne, Jean-Paul,Demeule, Michel
申请号：
AU2013273894
公开号：
AU2013273894A1
申请日:
2013.06.14
申请国别(地区):
AU
年份:
2015
代理人:
摘要：
The present invention is related to a compound that includes (a) α-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.
