Genes transferred into humans

Over 220 different genes have been introduced into cells in human gene therapy trials. It is impossible to discuss each gene in detail here, but, as would be expected, the gene types transferred most frequently match the most common group of diseases treated.

Cancer fighting genes
Cardiovascular diseases healing growth factors
Metabolics
Hiv
RNA interference

Cancer fighting genes
In around 60% of the trials the genes transferred are either genes coding for antigens used to stimulate an immune response, cytokine genes (cytokines are signalling molecules), tumor-suppressor genes or suicide genes, all of which are primarily used to combat cancer.

In 5.1% of trials of genes for receptors were used for cancer gene therapy.

In 2.1% of trials oncolytic viruses were transferred (rather than genes), these being aimed at destroying cancer cells.

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Cardiovascular diseases healing growth factors
Growth factors were transferred in 8.2% of trials, almost all of these being aimed at cardiovascular diseases.

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Metabolics
Deficiency genes were used in 7.9% of the trials in the fight against metabolic diseases.

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Diagnostics
Marker genes, meanly to diagnose, diseases were transferred in 4.1% of trials.

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Hiv
In 3.7% of trials replication inhibitors were used, to target HIV infection.

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RNA interference
1.7% of trials involved the transfer of antisense or short interfering RNA, with the aim of blocking the expression of a chosen gene.

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1. Michael L. Edelstein, Mohammad R. Abedi, Jo Wixon. Gene therapy clinical trials worldwide to 2007 - an update. Journal of Gene Medicine 2007;9(10): 833-842.

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Eelco Soeteman
Last updated december 3, 2007
This information is developed by Egan by order of Consert.
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