摘要 |
<p>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.</p> |
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 |