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
- Diagnostics
- HIV
- RNA interference
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 5.2% of trials of genes for receptors were used for cancer gene therapy. In 1.9% of trials oncolytic viruses were transferred (rather than genes), these being aimed at destroying cancer cells.
2. Cardiovascular diseases healing growth factors
Growth factors were transferred in 7.9% of trials, almost all of these being aimed at cardiovascular diseases.
Deficiency genes were used in 7.5% of the trials in the fight against metabolic diseases.
Marker genes, meanly to diagnose diseases, were transferred in 3.7% of trials.
In 4.4% of trials, replication inhibitors were used, to target HIV infection.
1.4% of trials involved the transfer of antisense or short interfering RNA, with the aim of blocking the expression of a chosen gene.