发明名称 |
TREATMENT OF SANFILIPPO SYNDROME TYPE B |
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. |
申请公布号 |
KR20130043166(A) |
申请公布日期 |
2013.04.29 |
申请号 |
KR20137001751 |
申请日期 |
2011.06.25 |
申请人 |
SHIRE HUMAN GENETIC THERAPIES, INC. |
发明人 |
CONCINO MICHAEL F.;CALIAS PERICLES;PAN JING;HOLMES KEVIN;MARTINI PAOLO;ROMASHKO ALLA;MEIYAPPAN MUTHURAMAN;ZHANG BOHONG;ISKENDERIAN ANDREA;LUNDBERG DIANNA;NORTON ANGELA;STRACK LOGUE BETTINA;HUANG YAN;ALESSANDRINI MARY;PFEIFER RICHARD |
分类号 |
A61K38/47;A61P25/00;A61P25/28 |
主分类号 |
A61K38/47 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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