Adenovirus vector for gene therapy on hemophilia B and application thereof

An adenovirus and hemophilia technology, applied in the field of adenovirus vectors, can solve the problems of complex packaging of adeno-associated virus and unsafe retroviruses

Inactive Publication Date: 2011-01-05
INST OF HEMATOLOGY & BLOOD HOSPITAL CHINESE ACAD OF MEDICAL SCI
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

[0004] The technical problem to be solved by the present invention is: aiming at the short-acting nature of simple adenoviruses, the unsafety of retroviruses, and the complexity of packaging of adeno-associated viruses, to provide a safe hemophilia drug that is integrated into the host genome at a fixed point. Adenoviral vector for B gene therapy and application thereof

Method used

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  • Adenovirus vector for gene therapy on hemophilia B and application thereof
  • Adenovirus vector for gene therapy on hemophilia B and application thereof
  • Adenovirus vector for gene therapy on hemophilia B and application thereof

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Embodiment Construction

[0022] The adenovirus vector used for gene therapy of hemophilia B of the present invention can simultaneously express hFIX and red fluorescent protein; each side of the hFIX and red fluorescent protein tandem gene expression box has a Cre integrase action site in the same direction Loxp site, inside the expression box there is the site-specific integrase PhiC31 action site attB.

[0023] There is an attB site between one of the Loxp sites and the target gene promoter, so that it can be integrated into the host genome in the presence of Phic31 integrase.

[0024] The loxp at the 5'end of the target gene expression cassette is adjacent to attB, forming a loxpattb fragment.

[0025] It can express red fluorescent protein, can be used as a tracer protein for adenovirus packaging, and is distinguished from traditional green fluorescent protein adenoviral vectors. When two adenoviruses work together with host cells, it can distinguish and trace.

[0026] It can express human coagulation fa...

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Abstract

The invention relates to an adenovirus vector for gene therapy on hemophilia B and application thereof, and the adenovirus vector can simultaneously express hFIX and red fluorescent protein; both sides of a tandem gene cassette of the hFIX and the red fluorescent protein are respectively provided with a loxp site of a Cre integrase action site in the same direction, and an action site attB of site-specific integrase PhiC31 is arranged in the gene cassette. The invention obtains a recombinant adenovirus vector which can be cyclized by DNA marked by the red fluorescent protein, and lays the foundation for the recombinant adenovirus vector to be further integrated into a main genome through attb sequences to exert the lasting function for treating hemophilia B. The adenovirus vector or adenovirus particles packaged outside the adenovirus vector is applied in the treatment of hemophilia B.

Description

Technical field [0001] The invention relates to an adenovirus vector, in particular to an adenovirus vector for treating hemophilia B that can be circularized and integrated into the host genome and its use. Background technique [0002] Hemophilia B is a hereditary bleeding disorder linked to the X sex chromosome. It is caused by a defect in the coagulation factor IX (FIX) gene. The main clinical manifestations are repeated spontaneous soft tissue and large joint bleeding. Repeated joint bleeding can cause Degenerative hemophilia arthropathy can cause teratogenesis and disability in severe cases. The severity of bleeding in patients with hemophilia B is related to the level of FIX in the circulation. At present, preventive factor replacement therapy has greatly reduced the frequency of bleeding and the occurrence of hemophilic joint diseases, and improved the survival and quality of life of patients with hemophilia, but patients still have fatal bleeding and chronic joint damage...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/861A61K48/00A61P7/04
Inventor 张磊杨仁池顾东生杜伟廷薛峰
Owner INST OF HEMATOLOGY & BLOOD HOSPITAL CHINESE ACAD OF MEDICAL SCI
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