Application of valeric acid derivatives in treatment of hereditary cerebellar ataxia

An ataxia, hereditary technology, applied in the prevention and improvement of hereditary cerebellar ataxia, the application of valeric acid derivatives in the treatment, autosomal recessive spinocerebellar ataxia 20, can solve the mechanism of SCAR20 Unclear, effective drug treatment is impossible to talk about, etc.

Active Publication Date: 2021-03-30
XIAMEN UNIV
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

At present, the mechanism of SNX14 mutation leading to...

Method used

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  • Application of valeric acid derivatives in treatment of hereditary cerebellar ataxia
  • Application of valeric acid derivatives in treatment of hereditary cerebellar ataxia
  • Application of valeric acid derivatives in treatment of hereditary cerebellar ataxia

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0105] Example 1. Construction and Identification of Conditional Knockout Mice in SNX14 Brain

[0106] Animals used in the experiment were used to construct SNX14 brain condition knockout mice using the Loxp-Cre system. f / f Nestin-CRE mice. Transgenic SNX14 involved flox / + (SNX14 f / + The mouse commissioning industry (Guangzhou) company was constructed, B6. CG-TG (NES-CRE) 1 kln / j (Nestin-Cre) mouse was purchased from Jackson Lab. First of all, SNX14 f / + Get SNX14 with Nestin-Cre f / + Nestin-cre mice, the latter is again with SNX14 f / f Matches to get the category knockout mouse SNX14 f / f Nestin-Cre figure 1 A), I can get SNX14 f / + SNX14 f / f SNX14 f / + Nestin-CRE mice. Four genotype mice can be identified by genotype identification of the child. figure 1 B). Immunoprotice verification, SNX14 f / f Nestin-Cre mouse central nervous system's brain regions did not express SNX14 ( figure 1 C) The peripheral administrator is expressed ( figure 1 D), indicating that the SNX14 gene is onl...

Embodiment 2

[0107] Example 2. Experimental process of VPA administration

[0108] The drug VPA was purchased from Sigma-Aldrich (P4543), with a 20 mg / kg spare with physiological saline (NS). Experiments are divided into three groups: SNX14 f / + ; Nestin-Cre + NS group, SNX14 f / f Nestin-Cre + NS group and SNX14 f / f Nestin-CRE + VPA group. Mice were administered from P25 days to receive abdominal injection VPA (250 mg / kg body weight / day) for 35 days, and the control group had a volume of NS ( figure 2 A). Three days (P63) after the end of the administration, behavioral tests were started. After the test was completed, the mice were killed, and the brain was subsequently experiment.

Embodiment 3

[0109] Example 3. VPA treatment has no significant effect on the body weight of mice.

[0110] See figure 2 B and 2C, during treatment (P25, P40, P50 and P60), SNX14 f / f Nestin-Cre + VPA group of mole weight and SNX14 f / f Nestin-Cre + NS groups have no significant differences, and females are also significantly different. It is suggested that the long-term VPA treatment has no significant adverse effect on the physical condition of the male and female. The data is indicated by mean ± standard mistake (Mean ± SEM), which was statistically analyzed by TW-WAY ANOVA, with 8-11 mice. NS representatives have no significant difference.

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PUM

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Abstract

The invention discloses an application of a compound shown in a formula I in preparation of medicines for preventing, treating or improving hereditary cerebellar ataxia, particularly autosomal recessive genetic spinal cerebellar ataxia 20(SCAR20). The compound provided by the invention can significantly improve the motor ataxia of a model mouse and slow down the death of Purkinje cells, thereby providing clinical support for the treatment of hereditary cerebellar ataxia, especially SCAR20.

Description

Technical field [0001] The present invention relates to the pharmaceutical field. Specifically, the present invention relates to the application of the valeric acid derivative in the treatment, preventing and improving hereditary cerebellar together, particularly the dyeing of the normally hidden genetic spinal cortex, 50 (SCAR20). Background technique [0002] Hereditary integration is a highly heterogeneous disease. The clinical manifestations are deteriorated in motion balancing, gait instability, eye movement and syndrome, and the incidence of approximately 8.9 / 100,000. According to genetic modes, it can be divided into normally stained, common stained hidden and X chain genetic. Among them, the incidence of common-stained hidden genetic integration is approximately 3 / 100,000, and patients show early sporting and coordination disorders, often accompanied by cerebellar atrophy or development. Demonstrative Body Recessive Genetic Spinal Cell Cerebral Culture Etus 20 (Autosoma...

Claims

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Application Information

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IPC IPC(8): A61K31/20A61P25/00A61P25/28A61P25/16
CPCA61K31/20A61P25/00A61P25/28A61P25/16
Inventor 王鑫张洪峰洪育娟杨伟杰王睿敏
Owner XIAMEN UNIV
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