Application of elemin in the preparation of medicines for treating lysosomal storage diseases

A technology of lysosomal storage disease and elemin, applied in the field of biomedicine, can solve the problems of easily causing other diseases, small benefits, and not being able to be used as a universal therapy for LSD
CN111671739BActive Publication Date: 2022-06-21ZHEJIANG UNIV
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Patent Information

Authority / Receiving Office
CN · China
Patent Type
Patents(China)
Current Assignee / Owner
ZHEJIANG UNIV
Publication Date
2022-06-21

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Abstract

The invention discloses the application of the small molecular compound elemin in the preparation of medicine for treating lysosomal storage diseases. In the present invention, by screening a small molecule library of natural extracts, the small molecule compound elemin is screened from 850 kinds of natural small molecules, which can effectively inhibit the phenotype of lysosomal storage diseases, for example, it can inhibit the volume of lysosomes increase, inhibit cholesterol storage, significantly restore inhibited lysosome tube formation, significantly restore tolerance to repeated starvation and reduce cell death rate, so as to achieve the purpose of reducing damage and inhibiting the development of the disease. Elemin has the prospect of being developed as a broad-spectrum drug for the treatment of lysosomal storage diseases.
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Description

technical field

[0001] The invention relates to the technical field of biomedicine, in particular to the application of elemenin in the preparation of medicines for treating lysosomal storage diseases. Background technique

[0002] Lysosomal storage disease (Lysosomal Storage Disease, LSD) is a general term for a class of inherited diseases caused by lysosomal-related single gene mutations. About 50 types of lysosomal storage diseases have been identified in humans, each with an average incidence of less than 1 in 100,000, but the total incidence is about 1 / 10,000 to 1 / 5,000.

[0003] The pathogenesis of LSD varies according to the mutated gene. The mutated genes can be enzymes responsible for degradation in lysosomes, proteins responsible for transporting substances on lysosomal membranes, proteins related to lysosomal membrane transport, and enzymes responsible for post-translational modification of lysosome-related proteins, etc. Although the pathogenesis is different, ...

Claims

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