new way to deal with congenital diseases French scientists discovered: with the help of the AIDS virus are introduced into the bone marrow of patients with children instead of inherited disease genes with the genes necessary for the treatment.
French scientists have been able to cure heavy congenital disease by introducing into the cells of the bone marrow for the treatment of adolescent desired genes. As a means of delivering gene DNA was used HIV - the virus infection of man that causes AIDS.
It is said that the best way of treatment - prevention. Of course, it is, in the case of acquired diseases, such as infections, parasitic and degenerative lesions. What if the child gets the disease from their parents? If the cause is not a way of life and bad habits and bad heredity and natural variability mechanisms. Most of the bacteria, viruses and parasites can be removed from the body, but how to change their own genes?
Until recently, genetically determined diseases able to treat only the symptomatic -podavlyat only some manifestations of the disease with drugs. However, I would like to learn how to treat the disease itself, in this case - to change the damaged genes.
This idea of gene therapy - treatment aimed at getting rid of the human genetic disease by introducing a normal gene of its body, so that they can overcome the effect of defective genes.
Once again the idea of a new method of treatment was borrowed from nature. After all, the ability to change the controlled and targeted a foreign gene have viruses embedded its genetic information into the host cells.
The more aggressive the virus - the more effective the treatment.
So decided researchers from the French National Biomedical agencies and used for the treatment of human immunodeficiency virus (HIV) as a carrier. It is this virus leads to one of the worst diseases - Acquired Immune Deficiency Syndrome (AIDS). Obur Patrick and Nathalie Cartier reported the results of treatment of congenital neurodegenerative disorders (adrenoleukodystrophy) two seven-year child at a meeting of the European Society of Gene and Cell Therapy.
Genetically determined diseases are caused by abnormalities in the structure of genes - the first stage of the classical scheme of "gene-protein-sign" that lead to the synthesis of defective protein, and consequently - the manifestation of the wrong sign.
A more sophisticated model looks like a "gene (DNA) - RNA - protein - modification of protein - a sign." To intervene in the development of congenital diseases can be at the level of DNA and RNA level, by way of which the protein is synthesized.
The most effective would be to prevent development of the disease at the earliest stages - in embryonic development, but the risk is extremely high stop scientists from such experiments, because in this case the change will have everything - including germ cells. And how will this affect the children cured person is still impossible to accurately predict.
Ongoing experimental and clinical research is based on bringing - adding a new gene to the existing defective. The targets are not all cells of the body, but only those tissues in which expression of this feature is most important - for example, nerve tissue in Parkinson's disease, or bone marrow in thalassemia.
Normal genes are introduced by various media - vectors. The most expensive but also the most effective method is the introduction of a normally functioning gene in a part of the adenovirus.
The same procedure can be repeated with greater reliability and ex vivo (outside the body) - collect cell tissue biopsy, to introduce them in a gene in vitro (in vitro) to control the efficiency, and then transplanted back. While minimizing possible side effects of the vectors that are not in contact with other cells of the organism.
So did Obur and Cartier, entering the correct gene with a vector of HIV in bone marrow cells in a cell culture, and then transplant them teenagers with the defeat of the shell of the nerve pathways reported Gazeta.Gazeta
By diseases treatable by gene therapy are not only innate - this method allows you to block proliferation of different viruses, or the formation of proteins that lead to the development of tumors. Among the most promising genetic disease therapy recessive disease when abnormal condition is caused by a recessive allele. In this case, adscititious gene at the DNA inhibits expression of the defective.
Despite the positive results and opened up prospects for the treatment of millions of people with congenital genetic diseases, not only the moralists, but scientists around the world do not hide their concerns about the possible negative impact of the viral component of "medicine" to human health.