What are genetics researchers studying and how can it help people with genetic disorders?
Now that a draft of the human genome map is complete, research is focusing on the function of each gene and the role that faulty genes play in disease. This will lead to improved diagnosis of diseases and a new approach to disease therapy. Researchers will create new classes of drugs based on gene sequencing and structure. These drugs, because they are targeted to specific sites in the body, will have fewer of the side effects common in many of today’s medicines. Other medications will be customized for an individual’s genetic profile. The potential for using genes themselves to treat disease – known as gene therapy – is the most exciting application of DNA science. This rapidly developing field has great potential for treating or even curing inherited and acquired diseases. Gene therapy will use normal genes to replace or supplement a defective gene, or to bolster immunity to disease. Currently, gene therapy research is primarily concerned with establishing the safety of this approach
