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METHODS AND COMPOSITIONS FOR MODULATING SIALIC ACID PRODUCTION AND TREATING HEREDITARY INCLUSION BODY MYOPATHY
专利权人:
STRIKE BIO; INC.
发明人:
Phillip B. Maples,Chris M. Jay,John J. Nemunaitis
申请号:
US15056929
公开号:
US20160175461A1
申请日:
2016.02.29
申请国别(地区):
US
年份:
2016
代理人:
摘要:
According to certain embodiments of the present invention, methods for modulating the production of sialic acid in a system are provided, which comprise providing the system with a wild-type GNE-encoding nucleic acid sequence. According to such embodiments, the system may comprise a cell, muscular tissue, or other desirable targets. Similarly, the present invention encompasses methods for producing wild-type GNE in a system that comprises a mutated endogenous GNE-encoding sequence. In other words, the present invention includes providing, for example, a cell or muscular tissue that harbors a mutated (defective) GNE-encoding sequence with a functional wild-type GNE encoding sequence.
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中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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