A TARGETED THERAPEUTIC FUSION PROTEIN

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专利权人:

发明人:

LEBOWITZ JONATHAN,MAGA JOHN

申请号：

IN2568/KOLNP/2006

公开号：

IN255138B

申请日:

2006.09.06

申请国别(地区):

IN

年份:

2013

代理人:

摘要：

Targeted acid alpha-glucosidase therapeutics that localize to the lysosome are provided. The targeted therapeutics include amino acid residues 70-952 of acid alphaglucosidase (GAA), and a peptide tag comprising a portion of IGF2, permitting proper subcellular localization of the targeted therapeutic upon internalization of the therapeutic. The targeted therapeutics are useful for remedying insufficient GAA activity in the lysosome, i.e., Pompe disease. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.

来源网站：

中国工程科技知识中心

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http://www.ckcest.cn/home/