Community Research and Development Information Service - CORDIS

Genetically engineered therapeutic cells

Recent and major technical advances in the field of biotechnology have allowed the rapid expansion of gene therapy. Manipulating and modifying genes within a single cell opens up a whole new world of methods for treating and curing a number of lethal diseases. The current research shows that it is possible to genetically alter cells of patients and transform them into producers of therapeutic antibodies.
Genetically engineered therapeutic cells
The defence mechanism against any disease is our immunity system. It uses highly specialised cells that are capable of identifying any potential infective agent and through the production of antibodies, these immune system cells finally neutralise the invader. Even though not all cells can act in the above-described way, ongoing research shows it is feasible to genetically alter cell id in order to transform them into defensive cells.

Gene therapy has already become a clinical reality and since its first successes its potential for providing therapy has been widely recognised. There are currently many ongoing human trials. Genetically engineering and altering the function of a cell is a powerful way for remedying genetic deficiencies and targeting specific tissues. Targeting mainly cancer and viral diseases, the current project has modified ex-vivo and in-vivo cells taken form a patient.

A sample of cells can be genetically engineered and modified ex-vivo to become a sample of cells now producing therapeutic monoclonal antibodies into the patient's bloodstream. Since the cells originate from the body of the patient they are not recognised as foreign invaders when, after being genetically altered, they re-enter the patient's bloodstream. In this manner a long-lasting and highly effective treatment is achieved.

Non-autologous antibody-producing cells, i.e. cells originating from a foreign host, can also be implanted by encapsulating them into immunoprotective devices. Such devices can be capsules made of cellulose sulphate. The capsules after insertion in the bloodstream form vascularised neorgans, stable over time, capable of both protecting the antibody-producing cells and allowing them to deliver the much-needed antibodies efficiently.

Yet another approach consists of genetically modifying patient's cells directly in-vivo using viral or non-viral vectors for the necessary gene transfer or alteration.

The therapeutic potential of these approaches was successfully tested experimentally on mice infected with the murine retrovirus that causes a lethal neurodegeneration. Patents protect these approaches and the results of the demonstration trials are available. The results from these processes cells will offer a lifelong protection against incurable cancers and viral diseases like AIDS.
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