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APPLICATION OF HSP70 AS AN ENZYMATIC ACTIVITY REGULATOR
专利权人:
ОРПХАСИМЕ АПС
发明人:
ЙЕНСЕН Томас Киркегор,ЙОТЕЛЛА Марья Хелена
申请号:
RU2014116069
公开号:
RU2014116069A
申请日:
2009.06.26
申请国别(地区):
RU
年份:
2015
代理人:
摘要:
1. The use of a bioactive agent capable of increasing the intracellular concentration of Hsp70 by enhancing the expression of the Hsp70 gene, characterized in that the bioactive agent is a hydroxylamine derivative for the treatment of lysosomal storage disease. The use of claim 1, wherein said bioactive agent is capable of enhancing Hsp70 gene expression under concomitant stress. The use of claim 1, wherein said hydroxylamine derivative is bimoclomol or a structural analog thereof. The use according to claim 1, wherein said hydroxylamine derivative is selected from the group consisting of arimoclomol, BRX-220, BRX-345, bimoclomol, BRLP-42 and BGP-15.5. The use according to claim 1, wherein said hydroxylamine derivative is arimoclomol (BRX-220 or BRX-345). 6. The use of claim 1, wherein said bioactive agent is formulated as a pharmaceutical composition further comprising a pharmaceutically acceptable carrier. The use of claim 1, wherein said treatment is prophylactic, therapeutic, healing or emollient. The use according to claim 1, wherein said lysosomal storage disease is selected from the group consisting of Nimann-Peak disease, Farber disease, Crabbe disease, Fabry disease, Gaucher disease, sialidosis, metachromatic leukodystrophy and saposin deficiency. The use according to claim 8, wherein said Nimann-Peak disease is selected from the group consisting of Nimann-Peak type A disease, Nimann-Peak type B disease, Nimann-Peak type C disease and Nimann-Peak disease type D.10. The use of claim 1, wherein said lysosomal storage disease is a lipid storage disease (or lipidosis). The application of claim 10, where the decree1. Применение биоактивного средства, способного повышать внутриклеточную концентрацию Hsp70 путем усиления экспрессии гена Hsp70, отличающееся тем, что биоактивное средство представляет собой производное гидроксиламина, для лечения лизосомной болезни накопления.2. Применение по п. 1, где указанное биоактивное средство способно усиливать экспрессию гена
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