RNA interference target for treating aids

Abstract

The present invention relates to RNA interference target sequence targeting HIV for the treatment of AIDS. Based on the target sequence, recombinant expression vectors, packaging vectors and cells were constructed, which express a siRNA and / or a miRNA and / or a ribozyme and / or an antisense oligonucleotide targeting HIV. Also provided is the use of the recombinant expression vectors, packaging vectors and recombinant cells in the manufacture of a medicament for the treatment of AIDS.

Description

FIELD OF THE INVENTIONThe present invention relates to molecular biology, cell biology and gene therapy. More specifically, it relates to 32 RNA interference (RNAi) targets which can be used for AIDS treatment, recombinant expression vectors using the targets, and drugs and methods for treating AIDS obtained in a variety of ways using these targets.BACKGROUND OF THE INVENTIONAcquired Immune Deficiency Syndrome (AIDS) caused by HIV infection is one of most significant health threat faced by the world. Currently, AIDS has almost spread to countries around the world, and resulted in more than 40 million patients suffering from it, and nearly 30 million people were killed by it (WHO, Report on the Global AIDS Epidemic, 2004). In recent years, the spread of AIDS in China is growing rapidly, and the infected people have already amounted to 0.84 million. At present, the treatment of HIV infection is primarily through high-intensity anti-retrovirus therapy, such as through a combined use of...

AI Technical Summary

Benefits of technology

In one embodiment, the recombinant expression vector of the present invention is characterized in that it comprises a nucleic acid sequence encoding the siRNA and / or the miRNA and / or the ribozymes and / or the anti-sense oligonucleotide of the RNA interference target sequence provided by the present invention, wherein the nucleic acid sequence is operably linked with the expression controlling sequence, making it possible to express the siRNA and / or the miRNA and / or the ribozymes and / or the anti-sense oligonucleotide targeting HIV in animal cells (especially mammalian cells, such as human cells, HIV receptor cells, preferably CD4+cells, such as mammalian stem cells, preferably hematopoietic stem cells).

The present invention also involves a cell carrying in or outside of its genome the nucleic acid sequence encoding the RNA interference target according to the present invention, including a prokaryotic cell (for example a bacterial cell, such as a E. coli cell) and a eukaryotic cell (such as a fungal cell, an insect cell, a plant cell, an animal cell, preferably a mammalian cell, such as a human cell, preferably a HIV receptor cell and a stem cell, such as a CD4+cell and a CD34+cell), which contains the nucleic acid sequence encoding the RNA interference target according to the present invention, wherein these nucleic acid sequences can be operably linked with the expression controlling sequence, making it possible to express the siRNA and / or the miRNA and / or the ribozyme, and / or the antisense oligonucleotide in the cell.

In a preferred embodiment, HIV receptor cells introduced with a shRNA expression element containing a nucleic acid sequence encoding the RNA interference target obtained from the present invention can thus acquire an ability to inhibit HIV replication and viral gene expression.

Problems solved by technology

However, due to the high mutation rate of HIV and its complex pathogenesis, this type of approach could not completely eradicate the virus in vivo.

However, due to the different inhibition efficiency of different targets, not all RNA interference targets met the requirement of conventional design are able to inhibit the expression of target genes effectively.

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