Priotities of Gene Therapy
Gene therapy is a relatively new area of medicine that attempts to apply recent advances in molecular biology, genetics and biotechnology to the treatment of human diseases. Gene therapy uses a set of approaches to the treatment of human disease based on the transfer of genetic material (DNA) into an individual. Gene delivery can be achieved either by direct administration of gene-containing viruses or DNA to blood or tissues, or indirectly through the introduction of cells manipulated in the laboratory to harbor foreign DNA. As a sophisticated extension of conventional medical therapy, gene therapy attempts to treat disease in an individual patient by the administration of DNA rather than a drug. (1)…show more content…
Their successful creation and introduction of an artificial human gene into the T cells of a young girl suffering from adenosine deaminase deficiency have brought to life many hopes for treating other genetic diseases. Among the first in that list were sickle-cell anemia, hemophilia, cystic fibrosis, muscular dystrophy, familial hypercholesterolemia (high serum cholesterol), and some cancers (melanoma, neuroblastoma, and brain tumors). The idea seemed simple and eloquent. Many inherited diseases are caused by a single faulty gene, and gene therapy would deliver the needed gene to a person's cells, which would then begin producing the missing essential substance. By 1995, there were 106 clinical trials (studies in humans) approved to test gene therapy for some of these diseases, and AIDS in the United States. (1) A number of impressive applications of the new recombinant DNA technology and the molecular pathology of single-gene disorders have been introduced.
The U.S. Government was providing annually about $200 million in research grants for gene therapy projects. To evaluate the effect of these investments, a special panel was set up by the National Institute of Health. Report and recommendations of the panel released in December 1995 summarized the recent progress of research in gene therapy. It also stated that the importance of this research for clinical