Key Points
Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight or prevent disease (see Question 2).
Researchers are studying gene therapy for cancer through a number of different approaches (see Question 3).
A gene can be delivered to a cell using a carrier known as a “vector.” The most common types of vectors used in gene therapy are viruses (see Question 4).
The viruses used in gene therapy are altered to make them safe; however, some risks still exist with gene therapy (see Questions 5 and 6).
A clinical trial using gene therapy must be approved by at least two review boards at the scientists’ institution, as well as by the U.S. Food and Drug Administration and the National Institutes of Health Recombinant DNA Advisory Committee (see Questions 9 and 10).
The Ethical, Legal, and Social Implications (ELSI) Research Program was established in 1990 to identify, analyze, and address the implications of human genetics research (see Questions 11 and 12).
What are genes?
Genes are the biological units of heredity. Genes determine obvious traits, such as hair and eye color, as well as more subtle characteristics, such as the ability of the blood to carry oxygen. Complex characteristics, such as physical strength, may be shaped by the interaction of a number of different genes along with environmental influences.
Genes are located on chromosomes inside cells and are made of deoxyribonucleic acid (DNA), which is a type of biological molecule. Humans have between 30,000 and 40,000 genes. Genes carry the instructions that allow cells to produce specific proteins, such as enzymes.
To make proteins, a cell must first copy the information stored in genes into another type of biological molecule called ribonucleic acid (RNA). The cell's protein synthesizing machinery then decodes the information in the RNA to manufacture specific proteins. Only certain genes in a cell are active at any given moment. As cells mature, many genes become permanently inactive. The pattern of active and inactive genes in a cell and the resulting protein composition determine what kind of cell it is and what it can and cannot do. Flaws in genes can result in disease.