Product used for treating and/or preventing beta hemoglobinopathy and fusion protein

A fusion protein and purpose technology, applied in the field of genetic engineering, can solve limited problems

Active Publication Date: 2021-06-18
EAST CHINA NORMAL UNIVERSITY +1
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  • Abstract
  • Description
  • Claims
  • Application Information

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  • Product used for treating and/or preventing beta hemoglobinopathy and fusion protein
  • Product used for treating and/or preventing beta hemoglobinopathy and fusion protein
  • Product used for treating and/or preventing beta hemoglobinopathy and fusion protein

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Embodiment Construction

[0055] In conjunction with following specific embodiment and accompanying drawing, the present invention is described in further detail, protection content of the present invention is not limited to following embodiment. Without departing from the spirit and scope of the inventive concept, changes and advantages that can be imagined by those skilled in the art are all included in the present invention, and are protected by the appended claims. The process, conditions, reagents, experimental methods etc. of implementing the present invention, except the content specifically mentioned below, are general knowledge and common knowledge in this field, and the present invention has no special limitation content. As described in Sambrook et al., Molecular Cloning, A Laboratory Manual (New York: Cold Spring Harbor Laboratory Press, 1989), or according to the manufacturer's suggested conditions.

[0056] 1. Working characteristics of fusion protein hyeA3A-BE4max

[0057] 1.1. Plasmid ...

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Abstract

The invention discloses a product used for treating and/or preventing beta hemoglobinopathy and a fusion protein. The fusion protein comprises a DNA binding structural domain of Rad51, a cytosine deaminase APOBEC3A mutant and nuclease, the cytosine deaminase APOBEC3A mutant means that asparagine at the 57th site of cytosine deaminase APOBEC3A is mutated into glycine. The product comprises a coding gene of the fusion protein and a delivery vector of sgRNA, and the sgRNA guides the fusion protein to carry out single-base gene editing on HBG1 and HBG2 promoter regions in target cells. According to the product used for treating and/or preventing the beta hemoglobinopathy and the fusion protein, the single-base gene editing efficiency can be greatly improved, and the HBG1 and HBG2 promoter region-117 sites are more accurately and efficiently targeted to generate G-to-A mutation, so that the expression of gamma-globin is activated, and a more accurate and efficient treatment strategy is provided for clinical treatment of beta-hemoglobinopathy.

Description

technical field [0001] The invention relates to the technical field of genetic engineering, in particular to a product and fusion protein for treating and / or preventing beta hemoglobinopathy. Background technique [0002] Since 2013, a new generation of gene editing technology represented by CRISPR / Cas9 has entered various experiments in the field of biology, changing the traditional means of gene manipulation. In April 2016, David Liu's laboratory first reported the single-base gene editing technology. After that, other types of single-base gene editing technology based on the principle of cytosine deaminase (such as cytosine deaminase from lamprey and human) Ammases are fused to dCas9 or Cas9n in different ways) have also been reported successively. It uses CRISPR / Cas9 derived from Streptococcus pyogenes (Streptococcus pyogenes) spCas9 uses NGG as PAM (spacer precursor adjacent motif) and recognizes and specifically binds DNA upstream of NGG to achieve a single base from ...

Claims

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Application Information

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IPC IPC(8): C07K19/00C12N15/62C12N15/113C12N15/85C12N5/10A61K38/50A61P7/06A61K38/46
CPCC12N9/78C12N9/16C12Y305/04001C12N15/113C12N15/85A61K38/50A61K38/465A61P7/06C07K2319/80C12N2310/10A61K2300/00
Inventor 李大力朱碧云张晓辉刘明耀
Owner EAST CHINA NORMAL UNIVERSITY
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