Minicircle gene vector and its preparation method and use

A gene carrier and micro-circle technology, which can be used in gene therapy, the introduction of foreign genetic material using vectors, and pharmaceutical formulations, etc., can solve the problem of low target cell efficiency.

Active Publication Date: 2013-01-23
SHENZHEN INST OF ADVANCED TECH CHINESE ACAD OF SCI
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  • Abstract
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  • Claims
  • Application Information

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Problems solved by technology

[0008] However, MC also has the problem of low delivery efficiency to target cells in vivo

Method used

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  • Minicircle gene vector and its preparation method and use
  • Minicircle gene vector and its preparation method and use
  • Minicircle gene vector and its preparation method and use

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Embodiment Construction

[0047] The microcircle gene carrier and its preparation method and application will be further explained below in conjunction with the accompanying drawings and examples.

[0048] A microcircle gene carrier is provided, and after the microcircle gene carrier is sent into target cells infected with viruses, viral gene therapy vectors can be produced. Target cells include hepatocytes in mammals, nonparenchymal hepatocytes such as liver endothelial cells, Kuffer cells or cells of other organs.

[0049] The produced viral gene therapy vectors can infect new target cells like wild viruses, produce new infectious recombinant viruses, and can express therapeutic transgene products themselves, so as to play a long-term and effective role and become a safe , Efficient, convenient and economical gene therapy technology.

[0050] This minicircle gene vector does not contain the backbone DNA components in standard plasmids that can turn off transgene expression, including: promoters, com...

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Abstract

The invention discloses a minicircle gene vector and its preparation method and use. The minicircle gene vector does not contain a backbone DNA ingredient which can close transgene expression and is in a standard plasmid. The minicircle gene vector comprises a promoter, an ingredient for gene expression and virus replication in a viral genome, and a transgene or a transgene expression box for expression of a treatment transgenosis product. The ingredient for gene expression and virus replication in a viral genome is cloned into a vector so that the combined chimeric minicircle gene vector is obtained. After being fed into a target cell infected by viruses, a small amount of the minicircle gene vectors can produce recombinant viruses having treatment and infection effects. Through infecting a new target cell, the recombinant viruses produce new recombinant viruses having infection effects, and finally, the recombinant viruses diffuse to all target cells. Compared with the traditional gene vectors, the minicircle gene vector can be efficiently delivered into a target cell and has strong and lasting treatment effects. The invention also provides a preparation method and a use of the minicircle gene vector.

Description

【Technical field】 [0001] The invention relates to the field of gene therapy, in particular to a microcircle gene carrier and its preparation method and application. 【Background technique】 [0002] Gene therapy is the most ideal way to treat many hereditary and acquired diseases, and there have been many successful reports in preclinical experiments. However, clinically successful cases are rarely reported. The core of gene therapy is to deliver therapeutic vectors into target cells, but there are still several technical problems to be solved in terms of vector construction and delivery. Gene therapy vectors can be divided into two categories, namely viral vectors and non-viral vectors. Viral vectors have a high efficiency of infecting cells, but due to their immunogenicity and insertional mutations, they are prone to cause severe reactions, including carcinogenicity and fatality, so they are difficult to be accepted clinically; in addition, high manufacturing costs are als...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/85C12N15/66A61K48/00A61P31/20A61P1/16
Inventor 陈志英何成宜
Owner SHENZHEN INST OF ADVANCED TECH CHINESE ACAD OF SCI
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