MOLECULES ABLE TO MODULATE THE EXPRESSION OF AT LEAST A GENE INVOLVED IN DEGRADATIVE PATHWAYS AND USES THEREOF
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United States of America Patent
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Issued Date -
Feb 16, 2012
app pub date -
Feb 11, 2010
filing date -
Feb 13, 2009
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Abandoned
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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.
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