Small molecule combination and application thereof

By using small molecule combinations to convert astrocytes into inhibitory neurons, the problem of association between GABAergic neuron dysfunction and neurodegenerative diseases in the prior art is solved, and efficient neuronal transformation and potential disease treatment effects are achieved.

CN119700761APending Publication Date: 2025-03-28JINAN UNIVERSITY
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Patent Information

Application Number
CN202311257237.3
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Filing Date
2023-09-27
Publication Date
2025-03-28

AI Technical Summary

Technical Problem

The prior art is difficult to effectively solve the association between GABAergic neuron dysfunction and a variety of neurodegenerative diseases, especially in the regenerative neuron technology, which has ethical challenges, high risks and high complexity.

Method used

A small molecule combination, including SB431542, LDN193189, CHIR99021, DAPT, Repsox, SAG and Purmorphamine, is provided for converting astrocytes into inhibitory neurons and in vivo transformation by in situ or intravenous injection.

Benefits of technology

The efficient transformation of astrocytes into functional inhibitory neurons was achieved, and significant transformation effects were demonstrated in vitro and in vivo, providing a potential direction for the treatment of neurodegenerative diseases.

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Abstract

The invention provides a small molecule combination. The small molecule combination comprises SB431542, LDN193189, CHIR99021, DAPT (Dimethylparaben-Amino Protein), Repsox, SAG (Soluble Adenosine Glycol) and Purmorphamine. The invention also provides application of the small molecule combination in preparation of drugs for converting astrocytes into neurons, application of the small molecule combination in preparation of drugs for treating nervous system diseases, and a method for converting astrocytes into neurons. The micromolecule combination provided by the invention can convert astrocytes into inhibitory neurons, and realizes in-situ transdifferentiation effect in a mouse body.
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