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Gene Therapy - Outlandish Sci-Fi or Medicine's Next Generation

Added: 02/06/2006

Wouldn't it be great if we could clone specific parts of our bodies? That way if one area in our body gets ill, we can swap it out with a disease-free substitute. In general there are many official and alternative methods of cancer treatment. Almost every day the scientists offer some new cancer therapy. One of the new and possibly most promising methods of cancer treatment is gene therapy. Clinical genetics is not sufficiently developed area and not all the fundamental laws and mechanisms of genetics are investigated. But there are some successive gene treatment trials and the main vectors in gene therapy are defined. Today there are several types of gene therapy.

There are many types of gene therapy nowadays. One of the most promising approaches to anticancer vaccine creation is a transfection of malignant cells with cytokine genes. After special adaptation and blockage these cells are able to live in the patient's organism and create cytokines that stimulate the antitumor immunity.

Russian Scientists from St. Petersburg Research Institute and Moscow Gene Biology Institute have developed the anticancer vaccine on the base of the patients? Malignant cells that were genetically modified. This technology was applied in 21 cases (17 patients with skin melanoma and 4 patients with metastatic kidney cancer). These trials were presented in Annals of Oncology.

The research proves that this gene therapy is nontoxic and the hyper-sensible reaction occurred in 48 per cent. The antitumor immune response was registered in 95 per cent. Full or partial regress was not registered. The stabilization of neoplastic process occurred in 8 cases (7 patients with skin melanoma and 1 patient with metastatic kidney cancer).
Scientists have decided that this method of gene therapy is promising in the cases of minimum tumor-after surgery.

This project is the first one in gene therapy that has reached the level of clinical trials.

Today these methods are used in gene therapy for tumors of different localization.

Proceedings of the National Academy of Sciences present the results of the research made by Medical center of Texas University. The scientists have made the trial when the mice with pigmentary xerodermia were effectively treated and the skin cancer development was prevented.

Pigmentary xerodermia is a serious hereditary disease. Usually children suffer from this skin disorder. The disease is caused by gene mutation. These genes are responsible for reparative process in the case of ultraviolet irradiation. People who suffer from pigmentary xerodermia must save their organisms from sunrays etc because they cause acute condition of the disease and stimulate skin cancer development. The risk of skin cancer development for people who suffer from this disease is 10000 times higher than this risk for usual people with unaffected skin.

The most frequent mutation is registered in gene XPA. This gene and neutralized virus were used as the genetic construction for the trial. The scientists have made the injection of gene XPA in affected skin of mice. These mice were irradiated by ultraviolet for several days. After five months the mice skin was healthy.

The scientists hope that medicine may use this technique for pigmentary xerodermia treatment and cancer prevention for the patients who have the mutation of gene XPA.


It is necessary to continue the research and trials for the mutation in other six genes that are responsible for the reparative processes in DNA.

So, today the most developed and promising vectors in gene therapy is anticancer vaccine on the base of malignant cells and a work with mutated gene that can stimulate the cancer development. The last method is the preventive one that is in general the most effective in all treatment areas.

A concept of science fiction? Not any more! Gene therapy is opening doors that we never could have imagined 20 years ago.




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