Molecules able to modulate the expression of at least a gene involved in degradative pathways and uses thereof

• 收藏
• 分享

专利权人:

Fondazione Telethon

发明人:

Ballabio, Andrea,Sardiello, Marco

申请号：

AU2013277001

公开号：

AU2013277001A1

申请日:

2013.12.30

申请国别(地区):

AU

年份:

2014

代理人:

摘要：

#$%^&*AU2013277001A120140220.pdf#####Abstract 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/