Treatment of sanfilippo syndrome type b
作者： Bettina Strack-Logue , Michael F. Concino , Pericles Calias , Richard Pfeifer , Angela Norton
DOI:
关键词: Biology 、 Lysosomal targeting 、 Intrathecal 、 Biochemistry 、 Moiety 、 Sanfilippo B 、 Molecular biology 、 Fusion protein 、 Sanfilippo syndrome 、 Recombinant DNA
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 a Naglu protein. A suitable protein can be recombinant, gene-activated or natural In some embodiments, is recombinant fusion containing domain lysosomal targeting moiety. an IGF-II
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