| abstract |
Provided are a method and a composition for treating Sanfilipo syndrome type B (Sanfilipo B). A method of treating Sanfilip syndrome type B comprising the step of intrathecally administering a recombinant α-N-acetylglucosaminidase (Naglu) protein to a subject in need of treatment, wherein the recombinant Naglu protein comprises a Naglu domain. And a lysosomal targeting moiety, further comprising a linker between the Naglu domain and the lysosomal targeting moiety. [Selection] Figure 1 |