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A base editing system, a method, a kit used for specificity repair for human HBB gene mutation and an application therefor

A base editing and specific technology, applied in the field of base editing system for specifically repairing human HBB gene mutation

Pending Publication Date: 2018-11-16
SUN YAT SEN UNIV
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  • Abstract
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Problems solved by technology

[0012] At present, the base editing system has been applied to single base gene editing in plants, yeast, human cells and mouse embryos 8-10 , but whether the base editing system can be used to repair HBB: c.-79A>G and HBB: c.-78A>G mutations, and treat β-thalassemia caused by the above mutations at the cellular level is still unknown

Method used

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  • A base editing system, a method, a kit used for specificity repair for human HBB gene mutation and an application therefor
  • A base editing system, a method, a kit used for specificity repair for human HBB gene mutation and an application therefor
  • A base editing system, a method, a kit used for specificity repair for human HBB gene mutation and an application therefor

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Embodiment Construction

[0144] The following descriptions are preferred implementations of the embodiments of the present invention. It should be pointed out that those skilled in the art can make some improvements and modifications without departing from the principles of the embodiments of the present invention. These improvements And retouching are also regarded as the scope of protection of the embodiments of the present invention.

[0145] Unless otherwise specified, the reagents and consumables used in the examples of the present invention are commercially available.

[0146]The practice of the present invention employs, unless otherwise indicated, conventional techniques of immunology, biochemistry, chemistry, molecular biology, microbiology, cell biology, genomics and recombinant DNA, which are within the skill of the art. See Sambrook, Fritsch, and Maniatis, MOLECULAR CLONING: A LABORATORY MANUAL, 2nd ed. (1989); Contemporary Molecular Biology Laboratory Manual (CURRENT PROTOCOLS IN MOLECUL...

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Abstract

The invention belongs to the field of gene treatment, and specifically relates to a method adopting a base editing system to repair beta thalassemia caused by A>G pathogenic mutation. The provided base editing system specifically repairs the above mutational method, mutational gRNA above specificity targeting and base editing protein. HBB: c.-79A>G and HBB: c.-78 A>G of human can be accurately repaired by utilizing the method. The method is high in efficiency and good in specificity. The gene editing system can be introduced into a somatic cell or individual of human, accurate repair can be performed on the A>G pathogenic mutation, thereby beta thalassemia disease can be cured, and the technology has wide application prospect in the field of gene treatment.

Description

technical field [0001] The invention relates to the fields of gene detection and gene modification, and relates to a base editing system, method, kit and application for specifically repairing human HBB gene mutations. Background technique [0002] Clustered regularly interspaced short palindromic repeat system (clustered regularly interspaced shortpalindromic repeat; CRISPR-associated, CRISPR-Cas) is an important immune defense system for archaea and bacteria to resist virus and plasmid infection, and is used to resist foreign genetic material. Invasions, such as phage viruses and foreign plasmids. At the same time, it provides acquired immunity for bacteria: this is similar to the acquired immunity of mammals. When bacteria are invaded by viruses or foreign plasmids, they will generate corresponding "memory" to resist their re-invasion. The CRISPR / Cas system can recognize foreign DNA or RNA and cut them off, silencing the expression of foreign genes. It is because of thi...

Claims

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Application Information

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IPC IPC(8): C12N15/113C12N15/90C12N15/63C12N5/10
CPCC12N15/113C12N15/63C12N15/907C07K14/4717C12N2310/10A61K48/00A61P7/00C12N5/10C12N15/90
Inventor 黄军就松阳洲梁普平孙宏伟张曦亚孙英
Owner SUN YAT SEN UNIV
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