Mika Aoyagi-Scharber,Teresa Margaret Christianson,Melita Dvorak-Ewell,Daniel J. Wendt,Shinong Long,Jonathan LeBowitz,Daniel Solomon Gold
申请号：
US14883193
公开号：
US09834587B2
申请日:
2015.10.14
申请国别(地区):
US
年份:
2017
代理人:
摘要：
The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases. Exemplary therapeutic fusion proteins comprise a lysosomal enzyme, a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. Also provided are compositions and methods for treating Mucopolysaccharidosis Type IIIB (Sanfilippo B Syndrome), comprising a targeted therapeutic fusion protein comprising alpha-N-acetylglucosaminidase (Naglu), a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide.
