发明公开
EP2218458A1 Molecules able to modulate the expression of at least a gene involved in degradative pathways and uses thereof
审中-公开
与能力分子以调节至少一种参与降解途径基因的表达及其用途
- 专利标题： Molecules able to modulate the expression of at least a gene involved in degradative pathways and uses thereof
- 专利标题（中）： 与能力分子以调节至少一种参与降解途径基因的表达及其用途
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申请号： EP09152778.8申请日： 2009-02-13
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公开(公告)号： EP2218458A1公开(公告)日： 2010-08-18
- 发明人: Ballabio, Andrea , Sardiello, Marco
- 申请人： Fondazione Telethon
- 申请人地址： Via Carlo Spinola 16 00154 Roma IT
- 专利权人： Fondazione Telethon
- 当前专利权人： Fondazione Telethon
- 当前专利权人地址： Via Carlo Spinola 16 00154 Roma IT
- 代理机构： Capasso, Olga
- 主分类号： A61K38/17
- IPC分类号： A61K38/17 ; C07K14/47
摘要：
A molecule 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 wherein said molecule acts on a CLEAR element; and/or on the TFEB protein activity or amount.
The molecule may be used in the treatment of neurodegenerative and/or lysosomal storage disorders.
The molecule may be used in the treatment of neurodegenerative and/or lysosomal storage disorders.
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