Gene Therapy

Overview

Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight or prevent disease. Genes, which are carried on chromosomes, are the basic physical and functional units of heredity.

Genes are specific sequences of bases that encode instructions on how to make proteins. Genes determine traits and characteristics from hair and eye color to the ability of the blood to carry oxygen. The interaction of a number of different genes in combination with environmental influences shapes many components of each person.

Although genes get a lot of attention, it's the proteins that perform most life functions and even make up the majority of cellular structures. When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result.

Image courtesy of The Genome Management Information System, Oak Ridge National Laboratory

 

Genes are located on chromosomes inside cells and are made of deoxyribonucleic acid (DNA), which is a type of biological molecule. Humans have between 21,000 - 25,000 genes.

Genes carry the instructions that allow cells to produce specific proteins such as enzymes.