Gene therapy carries the excitement of a cure-all for a host of diseases, the controversy surrounding the altering of human genes, and the promise of a type of medical treatment most of us would never imagine possible. With its potential to eliminate and prevent hereditary diseases such as cystic fibrosis and hemophilia and its use as a possible cure for heart disease, AIDS, and cancer, gene therapy is a potential medical miracle-worker.
But what about gene therapy for children? There’s a fair amount of risk involved in trials of this kind of therapy, and to date, only children who are seriously ill or have illnesses incurable by conventional means have been involved in clinical trials using gene therapy.
For those with serious illnesses that aren’t responsive to conventional therapies, however, gene therapy may soon offer hope that didn’t exist just a short time ago.
What Are Genes?
Your genes are part of what makes you unique. Inherited from your parents, they determine your physical traits – like the color of your eyes and the color and texture of your hair. They also determine things like whether you’ll be male or female, the amount of oxygen your blood can carry, and what your IQ will be.
Genes are composed of strands of a molecule called DNA and are located in single file within the chromosomes. The genetic message is encoded by the building blocks of the DNA, which are called nucleotides. There are approximately 3 billion pairs of nucleotides in the chromosomes of a human cell, and each person’s genetic makeup has a unique sequence of nucleotides. This is mainly what makes us different from one another.
Scientists believe that every human has about 25,000 to 35,000 genes per cell. A mutation, or change, in any one of these genes can result in a disease, physical disability, or shortened life span. These mutations can be passed from one generation to another, inherited just like a mother’s blond hair or a father’s brown eyes. Mutations can also occur spontaneously in some cases, without having been passed on by a parent. With gene therapy, the treatment or elimination of inherited diseases or physical conditions due to these mutations could become a reality.
Gene therapy involves the manipulation of genes to fight or prevent diseases. Put most simply, it introduces a “good” gene into a person who has a disease caused by a “bad” gene.
Two Types of Gene Therapy
There are two forms of gene therapy: