Small molecule polypeptide in HD treatment medicine and application of small molecule polypeptide

A technology of small molecule peptides and therapeutic drugs, which is applied in the field of biomedicine to achieve highly specific effects

Active Publication Date: 2021-07-09
NANJING MEDICAL UNIV
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

There is currently no effective treatment for the disease, and treatment options are limited to improving the symptoms of the disorder

Method used

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  • Small molecule polypeptide in HD treatment medicine and application of small molecule polypeptide
  • Small molecule polypeptide in HD treatment medicine and application of small molecule polypeptide
  • Small molecule polypeptide in HD treatment medicine and application of small molecule polypeptide

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Embodiment Construction

[0023] Below in conjunction with accompanying drawing and specific embodiment, further illustrate the present invention, should be understood that these embodiments are only for illustrating the present invention and are not intended to limit the scope of the present invention, after having read the present invention, those skilled in the art will understand various aspects of the present invention Modifications in equivalent forms all fall within the scope defined by the appended claims of this application.

[0024] Design and Screening of Small Molecule Peptides

[0025] like figure 1 As shown, use L-ALIGN sequence alignment software to determine Drp1

[0026] (dynamin-relatedprotein 1, mitochondrial dynamin-related protein 1) (human, NP_036192) and HSF1 (heat shock factor 1, heat shock transcription factor 1) (human, NP_005517) between the homology domain, sequence and position as figure 1 Shown in A. A truncated body with corresponding domain deletion was constructed an...

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Abstract

The invention relates to a small molecule polypeptide in an HD treatment medicine and application thereof. The amino acid sequence of the small molecule polypeptide is shown as SEQ ID No.1. The invention also provides application of the small molecule polypeptide. According to the invention, a small molecule polypeptide DH1 is designed by researching a translocation mechanism and functions of HSF1. The polypeptide achieves the purposes of protecting neurons and improving the HD disease progress by inhibiting mitochondrial translocation of HSF1, weakening mitochondrial fragmentation and improving the expression quantity of mitochondrial DNA, and provides a new target for HD treatment.

Description

technical field [0001] The invention relates to a small-molecule polypeptide in HD therapeutic medicine and its application, belonging to the technical field of biomedicine. Background technique [0002] Huntington's disease (HD) is an autosomal dominant genetic disorder caused by the expansion of the CAG repeat region in exon 1 of the huntingtin gene. While huntingtin (HTT) is expressed throughout the body, the polyglutamine-extended protein is particularly toxic to intermediate spiny neurons and their cortical connections in the striatum. The patient suffers from mood symptoms including depression and anxiety, as well as characteristic movement disturbances and chorea. There is currently no effective treatment for the disease, and treatment options are limited to improving the disorder. In striatal cell lines derived from HD transgenic mice, HD model mouse YAC128, fibroblast from HD patients, and iPSCs derived from HD patients, heat shock transcription factor 1 (HSF1) sh...

Claims

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Application Information

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IPC IPC(8): C07K14/47A61K38/17A61P25/14
CPCA61K38/00A61P25/14C07K14/4702
Inventor 郭兴付自醒刘纯月王晓嵩姜月晴
Owner NANJING MEDICAL UNIV
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