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Substances and Methods for the Treatment of Lysosmal Storage Diseases
专利权人:
Robert Steinfeld
发明人:
Robert Steinfeld
申请号:
US13515355
公开号:
US20120308544A1
申请日:
2010.12.14
申请国别(地区):
US
年份:
2012
代理人:
摘要:
The present invention relates to a chimeric molecule comprising (i) a targeting moiety that binds to heparin or heparan sulfate proteoglycans, (ii) a lysosomal peptide or protein, (iii) wherein the targeting moiety is a neurotrophic growth factor and/or, wherein the targeting moiety comprises one of the following consensus sequences BBXB, BXBB, BBXXB, BXXBB, BBXXXB or BXXXBB and wherein B represents an arginine, lysine or histidine amino acid and X represents any amino acid, (iii) with the proviso that the targeting moiety is at least thirteen amino acids long.
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