Molecules able to modulate the expression of at least a gene...

Details Molecules able to modulate the expression of at least a gene... Molecules able to modulate the expression of at least a gene...

A - Human Necessities

61

K

A61K 38/17 (2006.01) C07K 14/47 (2006.01)

Patent

CA 2752433

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.

L'invention concerne une molécule apte à moduler l'expression d'au moins un gène impliqué dans des voies de dégradation, de sorte à renforcer les voies de dégradation cellulaire et empêcher ou antagoniser l'accumulation de composés toxiques dans une cellule, et agissant sur un élément CLEAR. Les molécules préférées sont : la protéine TFEB et ses dérivés fonctionnels synthétiques ou biotechnologiques ; une molécule chimère contenant la protéine TFEB et ses dérivés fonctionnels synthétiques ou biotechnologiques ; un modulateur du niveau d'expression et/ou d'activité de la protéine TFEB. La molécule selon l'invention peut être utilisée dans le traitement des troubles neurodégénératifs et/ou des troubles du stockage lysosomal.

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