POSSIBILITIES AND RANGE OF GENE THERAPY

There is a growing body of evidence that the observed characteristics in pharmacokinetics and pharmacodynamics of drugs are genetically determined and that they primarily include drug metabolism and transport processes. Current acknowledgements, based on collected evidence, clearly show that an early adjustment of therapy regime to genetic characteristics of patients may help to avoid side effects. Such an approach stands for an individualized, optimal therapeutic use of drugs, based on the dose regime adjusted to an individual genotype. Gene therapy is the intentional transfer of genetic material into human somatic cells in prophylactic, therapeutic or diagnostic purposes. While the gene transfer technology is very advanced, ethical principles related to this procedure are still the subject of discussion. The goal of gene therapy is to correct genetic defects and to establish a normal cell functioning. Most of the techniques of gene therapy should enable the replacement of defective genes by those that function normally. The ideal vector should influence specific target cells, without stimulating the inflammatory response in the host cell. In addition, it should facilitate the transfer of genes of different length (the length of the code gene therapeutic sequences vary considerably and there should be a possibility of inserting regulatory sequences in transduction and expression) and integrate into the host cell chromosome at the exact location or to stay in the form of an episome within the nucleus (it should not be installed randomly in the chromosomes of the host, since it would mean a disturbed control of expression). The existing vector systems can be briefly divided into viral and non-viral. The paper presents the basic principles of the application of genes in the therapy of atherosclerosis, hereditary lung disease and cancer. Acta Medica Medianae 2011;50(3):74-80.