Basic group editing system and method for specifically repairing HBB gene mutation, kit and application of system and method and kit in human genital system

Abstract

The invention provides a basic group editing method for specifically repairing HBB gene mutation in a human genital system. The method comprises the steps of delivering a basic group editing system used for specifically repairing HBB gene mutation so that the basic group editing system is close to relevant sequences of HBB mutant genes, and obtaining repaired HBB genes, wherein the basic group editing system comprises a basic group editing enzyme and gRNA, the basic group editing enzyme is fusion protein, and the fusion protein includes effect protein structural domains, cytidine deaminase structural domains and uracil DNA glycosylase inhibitor structural domains of a CRISPR/Cas system. The gene editing system is guided into a human germ cell, a human fertilized ovum or a human embryo, A>Gpathogenic mutation can be precisely repaired, and thus beta-mediterranean anemia is cured. The basic group editing method has a wide application prospect in the field of genetic therapy.

Description

technical field [0001] The invention relates to the field of gene detection and gene modification, and relates to a base editing system, method, kit and application in human germline for specifically repairing human HBB gene mutation. Background technique [0002] The 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 against 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...

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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 level of cells, fertilized eggs or embryos still unknown

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