Looking for breakthrough ideas for innovation challenges? Try Patsnap Eureka!

Antisense oligonucleotide targeting ENSG00000203930 gene and application of antisense oligonucleotide

A technology of antisense oligonucleotides and antisense nucleotides, which can be used in genetic engineering, medical preparations containing active ingredients, recombinant DNA technology, etc., and can solve problems such as the development of targeted lncRNA molecules that are not applicable , to achieve an obvious effect

Active Publication Date: 2022-01-07
STAR PHARM (NANJING) INC
View PDF3 Cites 2 Cited by
  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0004] Since lncRNA does not produce protein products, the existing drug development methods for proteins, including small molecule inhibitors and biological macromolecular antibodies, are not suitable for the development of molecularly targeted drugs targeting lncRNA

Method used

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
View more

Image

Smart Image Click on the blue labels to locate them in the text.
Viewing Examples
Smart Image
  • Antisense oligonucleotide targeting ENSG00000203930 gene and application of antisense oligonucleotide
  • Antisense oligonucleotide targeting ENSG00000203930 gene and application of antisense oligonucleotide
  • Antisense oligonucleotide targeting ENSG00000203930 gene and application of antisense oligonucleotide

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0144] Example 1: Expression of ENSG00000203930 gene transcription in the human brain metastasis cancer cell line MDA-MB-231-BM Preparation of long-chain non-coding RNA transcript vector and the full sequence of the antisense strand of the transcript.

[0145] Through the extraction of RNA in MDA-MB-231-BM cells, reverse transcription, 3'-RACE, 5'-RACE, sequencing, generalist PCR, construction and preparation of expression vectors, and sequencing analysis, the long chain of the ENSG00000203930 gene was identified and disclosed Full sequence of the antisense strand of non-coding RNA transcripts. figure 1 The ENSG00000203930 gene transcription long-chain non-coding RNA transcript vector in the prepared MDA-MB-231-BM can be expressed in transfected cells. The sequence shown in SEQ ID NO.1 is the full antisense sequence of ENSG00000203930 gene transcription long-chain non-coding RNA product identified in the present invention in MDA-MB-231-BM, including 1,257 bases, of which A is ...

Embodiment 2

[0146] Example 2: Experimental ASO Design and Synthesis.

[0147] According to the long-chain non-coding RNA transcript sequence of the ENSG00000203930 gene identified in the present invention, a synthetic experiment ASO was designed. figure 2 The sequence showing the experimental ASO is CAAAGGCGCGGACTTA, a Gapmer containing a nucleotide modified with a 5'-phosphorothioate group and a locked nucleic acid. The synthetic ASO compound was identified by HPLC analysis and mass spectrometry, and its molecular weight was 5310.24Da.

Embodiment 3

[0148] Example 3: Experiment ASO inhibits the gene expression of ENSG00000203930 in cells.

[0149] By preparing the experimental ASO liposome, administering the experimental ASO dose as 50nM, and detecting the expression of the ENSG00000203930 gene in the cells by RT-qCP. image 3 It shows that compared with the control ASO, the experimental ASO can significantly inhibit the expression of the ENSG00000203930 gene in the cells, and the 50nM experimental ASO can reduce the expression of the ENSG00000203930 gene by about 50%.

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to View More

PUM

No PUM Login to View More

Abstract

The invention relates to an antisense oligonucleotide targeting an ENSG00000203930 gene and application of the antisense oligonucleotide and belongs to the technical field of gene drugs. The invention firstly identifies and discloses an antisense-strand complete sequence of a long-chain non-coding RNA transcript of the ENSG00000203930 gene and also discloses an antisense oligonucleotide sequence which is designed and synthesized on the basis of the antisense-strand complete sequence of the long-chain non-coding RNA transcript of the ENSG00000203930 gene. The invention also discloses application of the antisense oligonucleotide sequence in preparation of an ENSG00000203930 gene expression inhibitor. Research results show that the ASO synthesized or expressed on the basis of a vector can be used for remarkably inhibiting expression of the ENSG00000203930 gene, has an obvious brain metastasis inhibiting effect and has a clinical application prospect.

Description

technical field [0001] The invention belongs to the technical field of gene medicine, and in particular relates to an antisense oligonucleotide (ASO) targeting the ENSG00000203930 gene, a method for using the ASO to inhibit the expression of the ENSG00000203930 gene, and a method for treating diseases by administering a drug containing the ASO . Background technique [0002] Antisense oligonucleotide (ASO) regulation of specific target gene expression is derived from RNA interference technology, which is a technology that effectively regulates specific gene expression based on the principle of complementary base pairing. Through structural modification and transformation, including the modification and transformation of nucleotide backbones such as phosphorothioate substitution of 3', 5'-phosphodilipid bonds, and single nucleotide transformations such as substitution of locked nucleic acids, etc., greatly improved Druggability of ASO drugs. At present, a variety of ASO dru...

Claims

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to View More

Application Information

Patent Timeline
no application Login to View More
IPC IPC(8): C12N15/113A61K31/7105A61P35/00
CPCC12N15/1135A61K31/7105A61P35/00C12N2310/113
Inventor 陈礼明刘伟光何锡林
Owner STAR PHARM (NANJING) INC
Who we serve
  • R&D Engineer
  • R&D Manager
  • IP Professional
Why Patsnap Eureka
  • Industry Leading Data Capabilities
  • Powerful AI technology
  • Patent DNA Extraction
Social media
Patsnap Eureka Blog
Learn More
PatSnap group products

Browse by: Latest US Patents, China's latest patents, Technical Efficacy Thesaurus, Application Domain, Technology Topic, Popular Technical Reports.

© 2024 PatSnap. All rights reserved.Legal|Privacy policy|Modern Slavery Act Transparency Statement|Sitemap|About US| Contact US: help@patsnap.com