HBB gene kit for correcting autologous hematopoietic stem cell of patient suffering from servious beta-thalassemia
Patent Information
- Authority / Receiving Office
- CN · China
- Current Assignee / Owner
- 广东铱科基因科技有限公司
- Publication Date
- 2017-03-15
- Estimated Expiration
- Not applicable · inactive patent
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Abstract
Description
technical field
[0001] The invention relates to a gene therapy kit, in particular to an HBB gene kit for correcting autologous hematopoietic stem cells of severe beta-thalassemia patients. Background technique
[0002] β-thalassemia is a hereditary hemoglobinopathy, which belongs to autosomal dominant inheritance. It is a disorder of β-globin synthesis caused by mutation of β-globin gene (HBB). It is the most common monogenic genetic disease in the world.
[0003] The HBB gene is located in zone 1, zone 2, short arm of chromosome 11. Except for a few types of the disease, most of them are caused by point mutations (single nucleotide substitutions, additions or deletions). Mutations Those who cause partial inhibition of β chain synthesis are called "β + thalassemia"; those who cause complete inhibition of β chain are called "βo thalassemia". βo / βo homozygotes are severe, β+ / βo heterozygotes are mostly intermediate, and β+ / β is light. Due to the variety of β globin gene mutat...