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Transcription Factor Targeted Gene Therapy

BY: Nidhi Uppangala | Category: Others | Submitted: 2010-06-09 21:18:40
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Article Summary: "Human genome contains genes. sometimes genome also contains some genes which produce disease causing proteins. This disease causing gene can be replaced or inactivated using a method known as gene therapy by targeting the transcription factor protein..."


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Introduction:


Transcriptional factors are involved in the regulation of gene expression in our body. Human genome contains genes. These genes after tran script ion and translation produce proteins, these proteins are required for the normal functioning of the body. But sometimes genome also contains some genes which produce disease causing proteins. This disease causing gene can be replaced or inactivated using a method known as gene therapy.

Commonly used method of gene therapy is by using viral vectors functional gene is introduced into the human body. This method was successful in treating a condition known as X-linked severe immunodeficiency (X-SCID), genetic condition caused by the adenosine deaminase deficiency. Functional gene coding for adenosine deaminase is delivered into the patient's white blood cell using retrovirus vector.

Another method used in gene therapy is that the expression of a defective gene is altered by altering the transcription level of that gene. For example the expression of defective gene which codes for a disease causing protein, can be stopped by preventing the transcription of this defective gene into messenger RNA (mRNA) so that the defective protein is not produced by translation of gene. One of the new ways of performing this kind of gene therapy is by targeting the proteins involved in the gene transcriptions. In other words gene therapy is done by targeting the transcription factor proteins.

Tran script ion factor targeting gene therapy:

Tran script ion factor are proteins which regulate the expression of genes depending on the bodies need. These transcription factors regulated the activation and inactivation of genes in our body. Sometimes these transcriptional factors activate the genes which will produce disease causing proteins. In such cases the gene therapy can be used as cure. In such cases double stranded DNA strands or oligonucleaotides are introduced into the body. These DNA strands bind to the promoter region of the defective gene. By this the transcription factors will not bind to the promoter region of the defective gene which codes for the disease causing proteins. The DNA strands or oligonucleotides are introduced into the patient's body by using viral vectors. As our genome is very huge which contains many genes, as a result our body has got thousands of transcriptional factors. Scientists are continuously doing research to find out these transcription factors which can be used in gene therapy, to prevent the onset of diseases in human body.

Applications:

1. The DNA oligonucleotide targeting the transcription factor gene therapy is used to prevent the formation of lesions after vascular injury.

2. The DNA oligonucleotide targeting the transcription factor gene therapy is used to inactivate the tran script ion factor E2F.

3. The DNA oligonucleotide targeting the transcription factor gene therapy is also used against transcription factor known as NF-kB. These transcription factors are involved in the activation of genes which in turn produce cytokines and adhesion molecules. These molecules induce a disease condition known as myocardial infraction and glomerulonephiritis.

4. The DNA oligonucleotide targeting transcription factor gene therapy is also used to treat condition known as neointimal hyperplasia in vein bypass grafts.

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