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Medicine target point for treating friable X syndrome and application thereof in pharmacy

A syndrome and drug technology, applied to the drug targets for the treatment of Fragile X syndrome and its application in the field of pharmaceuticals, can solve the problems of infancy and no effective drugs, and achieve the effect of improving behavioral performance

Inactive Publication Date: 2010-01-06
FOURTH MILITARY MEDICAL UNIVERSITY
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

However, research on this as a drug target is still in its infancy
So far, there is no effective drug for the treatment of Fragile X Syndrome, and finding highly efficient and specific drugs has become an urgent task in today's pharmaceutical industry

Method used

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  • Medicine target point for treating friable X syndrome and application thereof in pharmacy
  • Medicine target point for treating friable X syndrome and application thereof in pharmacy
  • Medicine target point for treating friable X syndrome and application thereof in pharmacy

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0017] Example 1: Research on new drug targets using D1 receptor and GRK2

[0018] Materials and Methods

[0019] 1. Experimental animals

[0020] Adult (6-8 weeks) FMR1 knockout mice and wild-type normal control mice (FVB.129P2-Fmr1tm1Cgr strain) were purchased from Jackson Laboratories, USA. Raise in constant temperature, constant humidity, 12-hour lighting, free drinking animal room, feed pellet feed. All experimental procedures were in accordance with the regulations of the Animal Use and Management Committee of Fourth Military Medical University.

[0021] 2. Whole-cell Patch Clamp Recordings in Thin Sections of Adult Brain

[0022] Mice were anesthetized with 1-2% isoflurane, 300 μm ACC brain slices were cut transversely with a vibrating microtome, and placed in a mixed gas (95% O 2 and 5% CO 2 ) saturated artificial cerebrospinal fluid (ACSF), the experiment was performed after recovery for 1 h. ACSF composition (in mM): NaCl 124; NaHCO 3 25; KCl 2.5; KH 2 PO 4...

Embodiment 2

[0044] Example 2: Research on D1 receptor as a new drug target

[0045] The research method is the same as in Example 1. D1 receptor agonist SKF81297 was injected intraperitoneally for behavioral studies in mice.

[0046] Results: Intraperitoneal injection of D1 receptor agonist SKF81297 (1mg / kg) in mice can significantly inhibit the hyperactivity symptoms of FMR1 knockout mice. Significantly affected.

[0047] Conclusion: Behavioral studies have found that the use of D1 receptor agonists can improve the clinical manifestations of fragile X syndrome.

Embodiment 3

[0048] Example 3: Research on GRK2 as a new drug target

[0049] The research method is the same as in Example 1. Mouse behavior studies choose GRK2 inhibitor heparin intraperitoneal injection.

[0050] Results: Intraperitoneal injection of the GRK2 inhibitor heparin (1mg / kg) in mice can significantly inhibit the hyperactivity symptoms of FMR1 knockout mice. It can be seen that the total movement distance of the mice is significantly reduced at different times, but has no significant effect on the movement of normal mice .

[0051] Conclusion: Behavioral studies have found that blocking abnormal GRK2 activity in nerve cells can improve the clinical manifestations of Fragile X syndrome.

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Abstract

The invention relates to a novel medicine target point for treating friable X syndrome and an application thereof in pharmacy. The research discovers that the function of the FMR1 gene of a friable X syndrome model animal for knocking and removing a rat central nerve dopamine 1 type receptor (D1R) is lowered, and simultaneously discovers that the distribution of G-protein coupled receptor kinase-2 (GRK2) is abnormal, the expression of the GRK2 on a cell membrane is increased, and the GRK2 in cytoplasm is reduced. These pathological changes participate in the onset process of the friable X syndrome. A pharmacological method is adopted, an abdominal cavity injection D1R receptor excitant and a GRK2 suppressor are solely used or united, and the praxiology symptom of the friable X syndrome model animal can be obviously improved. The invention provides the novel medicine target point for treating the friable X syndrome and can develop novel treating medicines according to the target point, and the developed novel medicines not only can be solely used but also can be united to use.

Description

technical field [0001] The present invention relates to a drug target for the treatment of Fragile X Syndrome and its application in pharmacy, specifically, central nervous system dopamine type 1 receptor (D1 receptor) function reduction and / or G-protein coupled receptor kinase- The abnormally increased activity of 2 (GRK2) can be used as a drug target for treating fragile X syndrome, and can be used for preparing drugs for treating fragile X syndrome. technical background [0002] Fragile X syndrome is the most common inherited mental retardation. Its incidence rate is about one person in every 2000 males or 4000 females, and its clinical manifestation is mainly mental retardation (IQ is 40-60). According to conservative estimates, there are at least 400,000 Fragile X syndrome patients in my country. Fragile X syndrome is caused by the mutation of FMR1 (Fragile X Mental Retardation 1) gene, which leads to the lack of its encoded product FMRP protein in the body. The lack ...

Claims

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Application Information

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IPC IPC(8): A61K45/06A61K45/00A61P25/00
Inventor 招明高卓敏
Owner FOURTH MILITARY MEDICAL UNIVERSITY
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