Application of compound SS-31 to prepare medicine for treating Friedreich's ataxia and related diseases

A technology of SS-31, compound, applied in the field of application in the treatment of Friedreich's ataxia disease, to achieve the effect of huge market value and social benefit

Inactive Publication Date: 2017-11-07
NANJING UNIV
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

So far, there is no research on the regulation of SS-31 in diseases related to mitochondrial iron metabolism, let alone the research on FRDA, the disease I study

Method used

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  • Application of compound SS-31 to prepare medicine for treating Friedreich's ataxia and related diseases
  • Application of compound SS-31 to prepare medicine for treating Friedreich's ataxia and related diseases
  • Application of compound SS-31 to prepare medicine for treating Friedreich's ataxia and related diseases

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0058] Example 1 The optimization of SS-31 administration concentration and time is conducive to improving the expression of FXN in FRDA patient cells

[0059] 1.1 Experimental materials

[0060] Cells: lymphocyte line GM15850 derived from FRDA patients, normal human lymphocyte line GM15849. Culture conditions: culture in 1640 medium containing 10% fetal bovine serum (FBS), 2mM glutamate, and 100U / mL penicillin and streptomycin at 37°C and 5% CO2.

[0061] SS-31 was synthesized by Shanghai Qiangyao Biotechnology Co., Ltd., dissolved in PBS, stored at a concentration of 1mM, and stored at -80°C.

[0062] FXN antibody was obtained by immunization in our laboratory, and GAPDH antibody was purchased from Abgent.

[0063] 1.2 Experimental method

[0064] 1.1.1 Treat the patient's cells with 0, 2, 5, 10, 20, 50, 100, 200nM SS-31 respectively, collect the cell pellet and extract the protein after 24 hours. With healthy human cells as the control, after 24 hours of simultaneous cu...

Embodiment 2

[0069] Example 2 SS-31 Treatment Improves the Balance of Patient's Cellular Iron Metabolism

[0070] 2.1 Experimental materials

[0071] IRP2 antibody and TfR1 antibody were obtained by immunization in our laboratory, and Ferritin was purchased from Abcam.

[0072] Calcein-AM was purchased from sigma; RPA (Rhodamine B-[(1,10-phenanthroline-5-yl)-aminocarbonyl]benzyl ester) was purchased from Squarix.

[0073] 2.2 Experimental method

[0074] 2.2.1 First, detect the effect of SS-31 on the level of proteins related to iron metabolism in patients' cells. The patient's cells were treated with 50nm SS-31 for 8 and 24 hours, and the cell pellet was harvested. Western Blotting was used to detect the protein levels of IRP2, TfR1, Ferritin, ISCU, and FXN.

[0075]2.2.2 Secondly, the influence of SS-31 on the content of unstable iron in the patient's cytoplasm and mitochondria was detected. After treating the patient's cells with 50nmSS-31 for 8 and 24 hours, the cells were collecte...

Embodiment 3

[0078] Example 3 Effect of SS-31 treatment on the mitochondrial respiratory chain containing iron-sulfur clusters

[0079] 3.1 Experimental materials

[0080] Mitochondrial Complex I Activity Assay Kit (Abcam)

[0081] Mitochondrial Complex II Activity Detection Kit (Suzhou Keming Biotechnology Co., Ltd.)

[0082] Mitochondrial Complex III Activity Assay Kit (Biovision Inc.)

[0083] Xanthine Oxidase Assay Kit (Nanjing Jiancheng Technology Co., Ltd.)

[0084] 3.2 Experimental method

[0085] 3.2.1 First, the detection of aconitase activity: Prepare 8% separating gel according to the proportion (3.64mL of double distilled water, 0.94mL of 10×TB buffer solution, 1.68mL of 30% Acr-Bis, 22.5μL of 1M sodium citrate , 10% APS 31 μL, TEMED 6.25 μL), and mix the solution evenly. Take 5mL of separation gel and pour it into the gel-making device, then add 1mL of 50% ethanol to seal the liquid surface of the gel, pour off the ethanol after the separation gel is solidified, and then ...

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Abstract

The invention discloses an application of a compound Szeto-Schiller-31 (SS-31) to prepare a medicine for treating Friedreich's ataxia (FRDA) and related diseases. The invention belongs to the field of a medicine, and specifically relates to an application of SS-31 to treat FRDA diseases. It is found from experiments that SS-31 can up-regulate expression of frataxin (FXN) in FRDA patient's cells in a translational level, adjust iron metabolism in FRDA patient's cells, promote synthesis of iron-sulfur clusters in mitochondria, improve the function of mitochondria, reduce generation of ROS in patient's cells, and improve anti-oxidative stress performance of cells. The result shows that SS-31 has the potential value of treating FRDA.

Description

technical field [0001] The invention belongs to the field of medicine and relates to the application of compound Szeto-Schiller-31 (SS-31) in the treatment of Friedreich's ataxia (FRDA). [0002] Throughout this application, various publications are cited by first author and year of publication. Full citations for these publications are recorded in the References section at the end of the specification. The cited documents and publications, as well as the disclosures in the References section, are hereby incorporated by reference in their entirety into this application to more fully describe the state of the art as of the date of this invention. Background technique [0003] FRDA is a neurodegenerative disease caused by autosomal single-gene mutations and belongs to the category of mitochondrial diseases. The age of onset of the disease is often in early childhood, and the course of the disease develops progressively, and more than 40 to 50 years old die. The main clinica...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K38/07A61P25/28
CPCA61K38/07
Inventor 李宽钰
Owner NANJING UNIV
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