Application of Ptbp1 inhibitor in preventing and/or treating nervous system diseases related to functional neuron death

A neurological disease, neuron technology, applied in the field of biomedicine, can solve problems such as failure to prevent the development of the disease
CN112451669AInactive Publication Date: 2021-03-09CENT FOR EXCELLENCE IN BRAIN SCI & INTELLIGENCE TECH CHINESE ACAD OF SCI
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Patent Information

Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
CENT FOR EXCELLENCE IN BRAIN SCI & INTELLIGENCE TECH CHINESE ACAD OF SCI
Publication Date
2021-03-09
Estimated Expiration
Not applicable · inactive patent

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Abstract

The invention relates to an application of a Ptbp1 inhibitor in preventing and / or treating nervous system diseases related to functional neuron death. Specifically, the invention provides an application of a Ptbp1 gene or RNA or an encoded protein inhibitor of the Ptbp1 gene or RNA in preparation of a composition or a preparation, wherein the composition or the preparation is used for preventing and / or treating nervous system diseases related to functional neuron death. By inhibiting the expression or activity of the Ptbp1 gene or RNA or encoded protein of the Ptbp1 gene or RNA of astrocytes in the brain, transdifferentiation and differentiation of the astrocytes to dopamine neurons can be effectively induced; and meanwhile, by inhibiting the expression or activity of the Ptbp1 gene or RNAor encoded protein of the Ptbp1 gene or RNA in Muller glial cells in the retina, transdifferentiation of Muller glial cells into optic ganglion cells can be effectively induced, so that nervous system diseases related to functional neuron death are prevented and / or treated.
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Description

technical field

[0001] The invention relates to the field of biomedicine. More specifically, the present invention relates to the use of Ptbp1 inhibitors in the prevention and / or treatment of neurological diseases related to functional neuron death. Background technique

[0002] Parkinson's disease (PD) is a severe neurodegenerative disorder characterized by the loss of dopamine neurons in the substantia nigra of the midbrain. Previous studies have achieved direct reprogramming of astrocytes to dopamine neurons in vitro and in animal models by simultaneously overexpressing several transcription factors. However, Ptbp1-mediated neuronal reprogramming in vivo has not been reported so far.

[0003] At present, the main treatment for Parkinson's disease is the medicine represented by levodopa preparation. At the same time, surgical treatment can also improve symptoms to a certain extent. It should be pointed out that all these means can only partially alleviate the condition...

Claims

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