| 摘要 |
The present invention is related to a compound that includes a lysosomal enzyme (e.g., heparan sulfatase) and a targeting moiety, for example, where compound is a fusion protein including heparan sulfatase 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 lysosomal storage disorders (e.g., mucopolysaccharidosis Type IlIa) using such compounds. |
