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Lentiviral Vectors for Gene Therapy

Lentiviral Vectors for Gene Therapy

Gene therapy is expected to be an effective means for the treatment of genetic diseases, cancer, viral infections and other refractory disease, but the limitation of the gene transfer method is the biggest obstacle to achieve this hope.

Non viral gene transfer method has low efficiency. For the vast majority of gene therapies in human trials, virological methods are most used for gene transfer, and the retroviral vector and adenoviral vector are mature.

But the some antigens expression of adenovirus can cause the body's immune response, to prevent the repeated transduction. Some other viral vectors such as adeno-associated virus vector (AAV virus) and herpes simplex virus (HSV) vector are not satisfactory due to various reasons.

Recently, some researchers have focused on human immunodeficiency virus-1 (HIV-1) as the representative of the lentivirus. And this article will carry your attention to introduce the such vector.

The basic structure of HIV-1 genome

The main structure gene and arrangement form of HIV-1 DNA virus are the same as other retroviruses: 5 'LTR-gag-pro-pol-env-3' LTR. Different from other retroviral viruses, the HIV-1 genome has a lot of regulatory genes.

The basic principle of constructing HIV-1 carrier system

By co-transfection with 3 plasmids of packaging components and carrier components, the cells can harvest HIV-1 carrier particles, which only be infected by one time, can not be copied and carry target gene in the supernatant of the cells.

Improvement of HIV-1 carrier system

At present, for the HIV-1 carrier system in the field of gene therapy, the focus of research focused on how to expand the scope of its tropism, to ensure its safety and to provide its titer/transfer ability.

Existing problems

Despite the great progress in the research of HIV-1, there is still a long way to go in the clinical application.

First, the titer of the cre lentivirus is not high enough.

Secondly, due to the biological nature of HIV complex, it is difficult to establish a stable HIV vector package like the current mouse retroviral vector.