A gene is a part of DNA (deoxyribonucleic acid) sequences of nucleotides. Based on nucleotide sequences, some proteins are synthesized, which are the expression and function of cells and tissues. If there is a change in the gene sequence, protein synthesis is disturbed. In this condition, cells and tissues are not able, its normal function, resulting in genetic diseases.
What is Human Gene Therapy
Human gene therapy is a scientific method, a part of the nucleotide sequence or gene into a single cell for treatment of diseases. The most important principle of gene therapy is the return to normal functioning of cells and tissues by replacing abnormal or mutant genes. In simple terms, it is a method to correct defective genes. There are several ways to implement gene therapy a normal gene into the genome to replace a defective gene and a change in the regulation of gene expression. Another method of gene therapy, it is selective reverse mutation, so that the defective gene from its original form.
In most cases of human gene therapy a normal gene is inserted using a therapeutic vector or a carrier molecule. As its name suggests, the vehicle for the implementation of the normal gene on the target cell. Since the vector containing the therapeutic gene into the target cell (eg, liver cells), the gene therapy is beginning to produce functional proteins to restore normal function of target cells. While the Carrier or vector of the molecule, the different types of viruses such as retroviruses and adenoviruses are responsible for the end. Before using any of these viruses as vectors, the genome is contained by the elimination of disease genes and replaces it with the gene therapy.
Advantages and disadvantages of Human Gene Therapy
There are many possible side effects of using viruses as vectors, which are also some problems but, the inflammatory reaction of the immune system at the time, toxicity. A new technique for human gene therapy is the normal gene into the target cell without vectors. Although this method seems quite easy, it is not for all types of target cells. Another disadvantage of this method is that there is a large amount of DNA.
A major drawback of gene therapy is effective therapy in the short term after the introduction of gene into the target cell. It is from the cells in the body, which prevents the notion of therapeutic genes. After the insertion of foreign gene therapeutic target in the cell, it is necessary for the regulation of the self-reactions of the body. Human gene therapy is less effective for problems Multigenfamilien (presence of many defective genes).
Although the technology of human gene therapy is not as advanced in this case, it already has the scope of medical science. Like all genetic disorders, and most chronic diseases with the disorder of genes, gene therapy is a promising technique for the treatment of these serious health conditions. Genetic tests on the basis of human gene therapy are underway, options for treatment of chronic diseases like heart disease, cancer, cystic fibrosis and AIDS. Today, private grants and public subsidies, to the promotion of research on human gene therapy.