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Gene editing composition or kit for in vivo gene therapy

A technology of gene editing and composition, used in gene therapy, drug combination, genetic engineering, etc.

Active Publication Date: 2019-01-25
合肥星眸生物科技有限公司
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0005] In recent years, for genetic diseases such as muscular sclerosis, in vivo gene therapy has been successfully implemented in model animals. However, due to the limitations and optimizability of the method, it is difficult to target genetic diseases caused by gene defects in nerve cells. diseases, further development is needed to improve the efficiency of homologous recombination

Method used

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  • Gene editing composition or kit for in vivo gene therapy
  • Gene editing composition or kit for in vivo gene therapy
  • Gene editing composition or kit for in vivo gene therapy

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0083] Example 1 Design of sgRNA with MS2 targeting hairpin structure carrying specific targeting pde6b gene

[0084] In order to repair a nonsense mutation (TAC→TAA) on the seventh exon of pde6b in vivo and restore the function to a certain extent, a specific targeting pde6b gene was designed near this mutation site (Genbank NC_000071. 6) The sgRNA, the specific sequence of the sgRNA is SEQ ID NO 2.

Embodiment 2

[0085] Example 2 Design a template for homologous complementary repair. The template is a single-stranded DNA template with the normal sequence of the mutation site pde6b, and the specific sequence is SEQ ID NO 5.

Embodiment 3

[0086] Example 3 Construction of targeting MS2-recA fusion protein, the specific sequence is SEQ ID NO 10.

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PUM

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Abstract

The present invention provides gene editing compositions or kits for in vivo gene therapy. A gene editing composition or kit provided in the invention comprises 1) an sgRNA linked to a nucleic acid molecule of a binding protein targeting a mutant gene or a coding sequence thereof, 2) a template nucleic acid or code sequence thereof for repairing a mutant gene of inter, 3) a nuclease guided by sgRNA or coding sequence thereof, and 4) a protein fused with a nucleic acid bin protein capable of promoting homologous recombination or coding sequence thereof, wherein that nucleic acid binding proteinis capable of binding to the nucleic acid molecule in the above 1). The invention also relates to the use of sgRNA, compositions or kits for the treatment of diseases caused by gene mutations, such as hereditary diseases. The sgRNA, composition or kit of the invention can effectively improve the efficiency of homologous recombination repair, and can also realize homologous recombination repair innon-dividing cells.

Description

technical field [0001] The invention relates to the field of molecular biology gene editing. Specifically, the present invention uses a gene editing composition to efficiently and accurately treat gene mutations in vivo for diseases caused by gene mutations, such as hereditary diseases such as retinitis pigmentosa, hereditary muscular sclerosis, and hereditary tyrosinemia. Perform genetic repair. Background technique [0002] In recent years, due to the rapid development of genetic engineering technology, CRISPR / Cas9 technology has become one of the hot spots in the scientific community, and has been widely used in various in vivo and in vitro genetic transformation, construction of transgenic model animals, and gene therapy and other fields. Two important CRISPR technical papers were serialized in Science in 2013. The research group of Zhang Feng of MIT, the type II prokaryotic CRISPR adaptive immune system has been shown to promote RNA-guided site-specific DNA cleavage. ...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/113C12N9/22A61K48/00A61K31/7088A61P9/10A61P21/00A61P1/16A61P13/12A61P3/00
CPCA61K31/7088A61P1/16A61P3/00A61P9/10A61P13/12A61P21/00C12N9/22C12N15/113C12N2310/10C12N2310/20
Inventor 薛天仇子龙才源程田林
Owner 合肥星眸生物科技有限公司
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