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Lentivirus carrying erythroid gene editing system and medicine

A technology of gene editing and lentivirus, applied in the field of genetic engineering, can solve problems such as low efficiency, complicated operation, and poor safety performance, and achieve the effect of high efficiency, simple operation, and high safety

Active Publication Date: 2021-06-29
GENMEDICN BIOPHARMA INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

[0008] But at present, the method of electroporation is widely used at home and abroad to introduce the editing system of editing BCL11A enhancer into hematopoietic stem cells, which is complicated in operation, low in efficiency and poor in safety

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  • Lentivirus carrying erythroid gene editing system and medicine
  • Lentivirus carrying erythroid gene editing system and medicine
  • Lentivirus carrying erythroid gene editing system and medicine

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Embodiment Construction

[0044] To better explain the present invention, it will be described in detail by detail below with reference to the accompanying drawings.

[0045] In order to prepare a slow virus for the CRISPR-Cas9 gene editing system carrying a specific target Bcl11a enhancer, first construct a plasmid vector for implementing the relevant function, then plasmid vector and envelope protein, packaging plasmid co-transfected virus production Cells (such as 293T, 293FT or HEK293, etc.), were separated from supernatants after transfection, chromatography, to obtain a slow virus of the CRISPR-Cas9 gene editing system for delivering specific targets to BCL11A enhancers. One specific implementation method provided by the present invention is as follows:

[0046] First, plasmid construction

[0047] (1) Plenti-pHBB-Crispr-V2-H58-ENHACER2 plasmid vector construction:

[0048] The original promoter EF-1αCorePromoter in Plenti-Crispr-V2 (AddGene, # 78852) was replaced with a red-specific β-Globin, and th...

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Abstract

The invention relates to a lentivirus carrying an erythroid gene editing system. The lentivirus carries a sequence of a CRISPR-Cas9 gene editing system of a specific targeting BCL11A enhancer. According to the invention, the CRISPR-Cas9 gene editing system of the specific targeting BCL11A enhancer is delivered into hematopoietic stem cells through a lentiviral vector, BCL11A is inhibited in a targeting manner, and gamma-globin expression is reactivated, so that beta-thalassemia is relieved or cured. The lentiviral vector is used as a delivery system, and compared with an electroporation method commonly adopted at home and abroad, the lentiviral vector has the advantages of being high in infection capacity, easy to operate, high in efficiency and high in safety.

Description

Technical field [0001] The present invention relates to the field of genetic engineering, in particular, a slow virus and drug involving the editing system carrying a red gene. Background technique [0002] Mediterranean anemia and sickle cell anemia caused by hemoglobin to generate disorders due to mutant protein single gene mutation. Mediterranean anemia is the largest genetic disease in the world's largest distribution, the most incurred population, is also the largest disease in my country's most influence, the highest incidence rate, only about 30 million medlar anemia gene carriers, Mediterranean anemia in my country It is rare in northern China, but in Guangdong, Guangxi, Sichuan and other places. Sickle cell anemia is extremely low in my country, but in Africa's incidence is very high, because people carrying sickle cell anemia genetic mutation is not easy to infect malaria, it is not easy to infect AIDS, and Africa is the highest in malaria and AIDS. Region, therefore, p...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N7/01C12N15/867C12N5/10A61K48/00A61P7/06
CPCC12N7/00C12N15/86C07K14/47C12N5/0647A61K48/0058A61K48/0008A61P7/06C12N2740/15021C12N2740/15043C12N2310/20Y02A50/30
Inventor 董文吉张莹莹董祖伊张艳君刘子瑾程谟斌赵忠亮
Owner GENMEDICN BIOPHARMA INC
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