Michael F. Concino,Pericles Calias,Jing Pan,Kevin Holmes,Paolo Martini,Alla Romashko,Muthuraman Meiyappan,Bohong Zhang,Andrea Iskenderian,Dianna Lundberg,Angela Norton,Bettina Strack-Logue,Huang Yan,M
申请号：
US15715748
公开号：
US20180085438A1
申请日:
2017.09.26
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.
