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Molecules able to modulate the expression of at least a gene involved in degradative pathways and uses thereof
专利权人:
Fondazione Telethon
发明人:
Ballabio, Andrea,Sardiello, Marco
申请号:
AU2010212854
公开号:
AU2010212854B2
申请日:
2010.02.11
申请国别(地区):
AU
年份:
2013
代理人:
摘要:
A molecule being able to modulate the expression of at least a gene involved in degradative pathways so to enhance the cellular degradative pathways and prevent or antagonize the accumulation of toxic compounds in a cell and acting on a CLEAR element. Preferred molecules are: the TFEB protein, synthetic or biotechnological functional derivative thereof; chimeric molecule comprising the TFEB protein, synthetic or biotechnological functional derivative thereof; modulator of the TFEB protein activity and/or expression level. The molecule may be used in the treatment of neurodegenerative and/or lysosomal storage disorders.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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