发明名称 METHODS AND COMPOSITIONS FOR TREATMENT OF GLYCOGEN STORAGE DISEASES AND GLYCOGEN METABOLISM DISORDERS
摘要 The present disclosure provides for compositions comprising a chimeric polypeptide comprising a polypeptide effective for treating glycogen storage disease and an internalizing moiety that promotes delivery into cells. In certain embodiments, the polypeptide effective for treating glycogen storage disease is an acid alpha-glucosidase (GAA), a laforin, an amyloglucosidase (AGL), a malin, or an alpha amylase. The present disclosure also provides for methods for decreasing glycogen accumulation in cells or for treating glycogen storage diseases, including Forbes-Cori Disease, Andersen Disease, von Gierke Disease, Pompe Disease, and Lafora Disease, comprising administering the chimeric polypeptide disclosed herein.
申请公布号 WO2015192092(A1) 申请公布日期 2015.12.17
申请号 WO2015US35680 申请日期 2015.06.12
申请人 VALERION THERAPEUTICS, LLC 发明人 ARMSTRONG, DUSTIN, D.
分类号 A61K38/47;A61K39/395;A61P3/08;C07K14/47;C07K19/00 主分类号 A61K38/47
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