Treatment of sanfilippo syndrome type b by intrathecal administration of alpha-n-acetylglucosaminidase
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United States of America Patent
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Nov 14, 2017
Grant Date -
Nov 7, 2013
app pub date -
May 10, 2013
filing date -
Jun 25, 2010
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Abstract
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.
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