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

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 ...

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

Images