Aoyagi-Scharber, Mika,Christianson, Teresa Margaret,Dvorak-Ewell, Melita,Wendt, Daniel J.,Long, Shinong,Lebowitz, Jonathan,Gold, Daniel Solomon
申请号：
AU2013352184
公开号：
AU2013352184A1
申请日:
2013.11.27
申请国别(地区):
AU
年份:
2015
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.
