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Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders

机译：治疗糖原贮积病和糖原代谢异常的方法和组合物

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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.

机译：本公开内容提供了包含嵌合多肽的组合物，所述嵌合多肽包含有效治疗糖原贮积病的多肽和促进递送到细胞中的内化部分。在某些实施方案中，有效治疗糖原贮积病的多肽是酸性α-葡糖苷酶（GAA），laforin，淀粉葡糖苷酶（AGL），苹果酸或α淀粉酶。本公开还提供了用于减少细胞中糖原积累或用于治疗糖原储存疾病的方法，包括福布斯-科里病，安德森病，冯·吉尔克病，庞贝病和拉福拉病，包括施用本文公开的嵌合多肽。

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公开/公告号US10781434B2

专利类型
公开/公告日2020-09-22

原文格式PDF
申请/专利权人 VALERION THERAPEUTICS LLC;
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申请/专利号US201916273095
发明设计人 DUSTIN D. ARMSTRONG;
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申请日2019-02-11
分类号C12N9/24;C12N9/34;C07K16/44;A61K38/46;C12N9/26;A61K47/68;C12N9/16;A61K38/47;A61K39;A61K38;
国家 US
入库时间 2022-08-21 11:30:36

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