The Therapeutic Potential of Gene Therapy

Gene therapy, a new treatment, is to transfer nucleic acid (DNA or RNA) into selected somatic cells (somatic cells) to correct genetic defects and produce therapeutic proteins. It can be done ex vivo ("away from life") or in vivo ("in vivo"). In the ex vivo method, the target cells are removed from the patient, genetically modified, and reintroduced into the patient. This method is effective, but it is limited to easily accessible cells such as epithelial cells (cells covering the inner and outer surfaces of the body), muscle cells, blood cells, stem cells and so on.

In somatic gene therapy (SCGT), therapeutic genes are introduced into any cell other than gametes, germ cells, gametophytes or undifferentiated stem cells. Such modification affects only individual patients and not to descendants. Somatic gene therapy represents the mainstream and clinical studies where therapeutic DNA (integrated into the genome or as an external episome or plasmid) is used to treat the disease. Over 600 clinical trials using SCGT are being conducted in the United States. Most people are concerned about serious genetic diseases such as immunodeficiency, hemophilia, thalassemia, cystic fibrosis. This single gene disorder is a good candidate for somatic cell therapy. It is still impossible to completely correct genetic diseases or replace multiple genes. There are only a few studies in the late stage

v. Gene therapy is a method of treating genetic diseases (or acquired diseases) by replacing abnormal genes with therapeutic genes. As with Tay-Sachs disease, cystic fibrosis and other diseases can be cured by gene therapy. Currently, biotechnology experts are also conducting experiments using gene therapy to treat tumors, cancers and others. The resulting change is also conveyed to the next generation, ie in the case of germline gene therapy, these changes are hereditary. Another type of gene therapy is somatic cell gene therapy. It involves correcting genetic defects by introducing therapeutic genes into the somatic cells of the body. The changes that occur in this way are not hereditary