A product for the treatment of hemophilia b

Abstract

The present application discloses a product for treating hemophilia B. The product includes: a reagent capable of inserting a target gene into intron No. 13 of Alb (Albumin, albumin) in hepatocytes and co-expressing the target gene and Alb; the target gene includes F9 factor coding gene. In this application, the 13th intron of the Alb gene in hepatocytes is selected as the insertion site of the exogenous gene for the first time, and the integrated donor sequence designed according to this site can ensure the complete expression of Alb and co-expression with the exogenous gene, thereby achieving The purpose of high expression of exogenous genes is achieved without destroying the expression of endogenous genes. The product provided by the present application can improve the treatment and prevention effects of hemophilia B, has higher efficiency and better safety, and has a good application prospect in clinical treatment.

Description

technical field [0001] The invention relates to the field of gene therapy, in particular to a product for treating hemophilia B. Background technique [0002] Many genetic diseases are caused by genetic mutations in certain metabolism-related proteins expressed in the liver. These mutated proteins often lose their function, resulting in the inability of related metabolic processes or normal physiological and biochemical processes, resulting in diseases such as hemophilia A, hemophilia B, tyrosinemia, and phenylketonuria. The existing therapeutic mechanisms are mostly protein replacement therapy, liver transplantation, and gene therapy in the form of gene supplementation. [0003] Protein replacement therapy and small molecule drug therapy are often expensive and require long-term treatment, which is a heavy burden on the patient's body and economy. For liver transplantation, it also faces several major problems of insufficient donors, surgical risks and immune rejection. ...

AI Technical Summary

Problems solved by technology

[0011] 3. Choose homologous recombination to integrate exogenous gene fragments. Knock-in (integration) cannot be achieved in non-dividing cells. There are restrictions on the type of therapeutic cells. It is only suitable for the treatment of newborn mice, and the existence of long homologous arms Limits the loading capacity of AAV viruses to exogenous CDS

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