PROGRANULIN (PGRN) AND ITS DERIVATIVES FOR DIAGNOSIS AND TREATMENT OF LYSOSOMAL STORAGE DISEASES

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专利权人:

Chuanju Liu;Jinlong Jian

发明人:

Chuanju Liu,Jinlong Jian

申请号：

US15116122

公开号：

US20170049855A1

申请日:

2015.02.04

申请国别(地区):

US

年份:

2017

代理人:

摘要：

The present invention provides composition and methods for diagnosis and treatment of lysosomal storage diseases and their diagnosis and treatment, including Gaucher's Disease and Tay-Sachs disease, and particularly which utilize progranulin (PGRN), or active PGRN peptides, including atsttrin. The invention also provides animal models of lysosomal storage diseases, including Gaucher's Disease and Tay-Sachs disease, based on or including PGRN mutations including PGRN null mutants and PGRN gene knock outs.

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中国工程科技知识中心

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