Thalassemia-induced multipotent stem cell as well as preparation method and application thereof

A technology for pluripotent stem cells and thalassemia, which is applied in the field of thalassemia-induced pluripotent stem cells and their preparation, and can solve problems to be improved, etc.

Active Publication Date: 2012-08-08
GUANGZHOU INST OF BIOMEDICINE & HEALTH CHINESE ACAD OF SCI
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  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

[0004] However, the current induced pluripotent stem cell technology, especially the...

Method used

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  • Thalassemia-induced multipotent stem cell as well as preparation method and application thereof
  • Thalassemia-induced multipotent stem cell as well as preparation method and application thereof
  • Thalassemia-induced multipotent stem cell as well as preparation method and application thereof

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Experimental program
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Embodiment 1

[0051] 1. The acquisition and identification of original induced pluripotent stem cells

[0052] First, exosomes containing cDNA or miRNA clusters of pluripotent stem cell-inducing factors were introduced into the amniocytes of thalassemia patients by nucleofection method, wherein, compared with the normal β-globin gene, the thalassemia patients had The two alleles of the β globin gene in the amniocyte genome had the same mutation, and the mutation type was IVS-H-654; the pluripotent stem cell induction factors used were Oct4, Sox2, Nanog, Lin28 and miR- A combination of 302-367.

[0053] Then, the amniotic fluid cells introduced with pluripotent stem cell-inducing factors were directly transferred into a serum-free, cell-free feeder layer human embryonic stem cell culture environment, and after 20 days of culture, a large number of typical primary induced pluripotent stem cell clones were obtained, and these clones were picked Then continue to cultivate. Two obtained origin...

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Abstract

The invention relates to a thalassemia-induced multipotent stem cell as well as a preparation method and application thereof. The multipotent stem cell is derived from a body cell of a thalassemia patient; and a genome of the thalassemia-induced multipotent stem cell contains a normal beta globin gene. The thalassemia-induced multipotent stem cell disclosed by the invention can be differentiated into hemopoietic stem cells under proper conditions; and the hemopoietic stem cells can express the normal beta globin gene.

Description

technical field [0001] The present invention relates to the field of biomedicine. Specifically, the present invention relates to thalassemia induced pluripotent stem cells and their preparation methods and uses. Background technique [0002] Thalassemia (thalassemia, thalassemia) is a group of the most common monogenic genetic diseases in the world. It is a disease in which hemoglobin (Hb) function is abnormal due to globin gene defect, and it is manifested as chronic progressive hemolytic anemia. Thalassemia has a very high incidence among people in Guangdong, Guangxi and other places in my country. At present, it mainly relies on regular blood transfusion for maintenance treatment, which brings a heavy burden to patients and society. Among them, the treatment of β-thalassemia major is more difficult. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is currently the only clinical means to cure β-thalassemia major. However, as we all know, the source of hema...

Claims

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Application Information

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IPC IPC(8): C12N5/10C12N5/074C12N15/85C12N5/0789A61K35/22A61K35/14A61K35/36A61K35/50A61K35/44A61P7/06C12Q1/02A61K35/12A61K35/28
Inventor 潘光锦裴端卿廖宝剑马宁
Owner GUANGZHOU INST OF BIOMEDICINE & HEALTH CHINESE ACAD OF SCI
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