AOYAGI-SCHARBER, Mika,CHRISTIANSON, Teresa,DVORAK-EWELL, Melita,WENDT, Daniel,LONG, Shinong,LEBOWITZ, Jonathan,GOLD, Daniel
申请号:
HUE13802834
公开号:
HUE039334T2
申请日:
2013.11.27
申请国别(地区):
HU
年份:
2018
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.