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Application of DHCR24 gene in preparation of medicine for treating neurodegenerative diseases

A DHCR24, neurodegenerative technology, which is applied in the application field of DHCR24 gene in the preparation of drugs for the treatment of neurodegenerative diseases, can solve the problems of low transfection efficiency, cumbersome manufacturing process of immunogenic carcinogenesis, etc., and achieves enhanced synthesis and supply , Correct the lack of cholesterol in the brain, improve the effect of learning and memory function

Pending Publication Date: 2022-05-20
JINSHAN HOSPITAL AFFILIATED TO FUDAN UNIV (EYE DISEASE PREVENTION & TREATMENT CENT OF JINSHAN DISTRICT RES CENT FOR CHEM INJURY EMERGENCY & CRITICAL MEDICINE OF SHANGHAI MUNICIPAL HEALTH COMMISSION)
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  • Description
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AI Technical Summary

Problems solved by technology

AAV viruses have been used as gene therapy vectors and are more efficient in cellular uptake, gene integration, and long-term gene expression, especially in vivo, but they are immunogenic, potentially carcinogenic, and cumbersome to manufacture
To address these limitations, nonviral vectors capable of avoiding immune responses, broadly regulatable, and quality-controlled have been developed, but they suffer from low transfection efficiency and lead to passive expression of transgenes
In conclusion, its clinical application is still limited by gene carrier immunogenicity, carcinogenicity, off-target effects and efficacy

Method used

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  • Application of DHCR24 gene in preparation of medicine for treating neurodegenerative diseases
  • Application of DHCR24 gene in preparation of medicine for treating neurodegenerative diseases
  • Application of DHCR24 gene in preparation of medicine for treating neurodegenerative diseases

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Embodiment 1

[0037] The technical contents of the present invention are as follows: 1) firstly, for the target target gene DHCR24, the design and construction of the target gene are carried out, and the mouse DHCR24 (mouse-DHCR24, m-DHCR24) gene overexpression adeno-associated virus 9 (AAV9) vector is completed. Construction (technical assistance provided by Hanbio Technology (Shanghai) Co., Ltd.), and finally the preparation of AAV9-wrapped DHCR24 gene drug (that is, AAV9 vector complex containing DHCR24 gene). 2) We will transfect the prepared AAV9 drug carrying DHCR24 gene (AAV9 package DHCR24 gene drug) into specific nerve tissue (or cell) through AAV9-mediated DHCR24 gene transfection method to improve specific nerve tissue (or cell) ) DHCR24 expression level, thereby increasing the synthesis of cholesterol in nerve tissue (or cells) and increasing the supply of cholesterol, and helping to correct the technical method of cholesterol deficiency (lack) in nerve tissues (or cells).

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Abstract

The invention relates to an application of an AAV9 carrier compound containing a DHCR24 gene in preparation of a medicine for treating neurodegenerative diseases. The neurodegenerative diseases are Alzheimer's disease (AD), Parkinson's disease, brain senescence, cognitive disorder related to senescence, autism, Niemann-Pick's disease, Huntington's disease, motor neuron disease, frontotemporal dementia, dementia with Lewy bodies, multiple sclerosis and Rett syndrome. The invention also provides a medicine for treating neurodegenerative diseases. On the basis of a new cholesterol theory of AD morbidity, synthesis and supply of cholesterol in the brain are enhanced through a DHCR24 gene treatment method (DHCR24 gene function enhancement), and deficiency (or deficiency) of cholesterol in the brain is helped to be corrected, so that the learning and memory functions of AD are improved.

Description

technical field [0001] The invention relates to the technical field of gene therapy, in particular to the application of the DHCR24 gene in the preparation of drugs for treating neurodegenerative diseases. Background technique [0002] Alzheimer's disease is a degenerative disease that is more common in dementias, accounting for more than 60% of them. Clinically, memory impairment is the most common, characterized by the overall decline of the patient's cognition, behavior, language and other brain function activities, and it is irreversible. [0003] 24-Dehydrocholesterol reductase (DHCR24) is a key gene that controls cholesterol synthesis in cells: Studies have found that the 24-Dehydrocholesterol reductase (24-Dehydrocholesterol reductase, DHCR24) gene is about 46.4kb in length and located on chromosome 1 1p31.1 -p33, containing 9 exons and 8 introns, the gene encodes a protein molecule with 516 amino acid residues. In the cellular cholesterol synthesis pathway, DHCR24 ...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K48/00A61P25/28A61P25/00A61P25/16A61P25/14
CPCA61K48/005A61K48/0008A61P25/28A61P25/00A61P25/16A61P25/14
Inventor 祖恒兵
Owner JINSHAN HOSPITAL AFFILIATED TO FUDAN UNIV (EYE DISEASE PREVENTION & TREATMENT CENT OF JINSHAN DISTRICT RES CENT FOR CHEM INJURY EMERGENCY & CRITICAL MEDICINE OF SHANGHAI MUNICIPAL HEALTH COMMISSION)
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