KidsHealth for Parents – Gene Therapy and Children

Gene therapy carries the promise of cures for many diseases and for types of medical treatment most of us would not have thought 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 kids 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.

Our genes are part of what makes us unique. Inherited from our parents, they go far in determining our physical traits like the color of our eyes and the color and texture of our hair. They also determine things like whether babies will be male or female, the amount of oxygen blood can carry, and IQ.

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. Approximately 3 billion pairs of nucleotides are 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 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 red hair or a father's brown eyes. Mutations also can 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 simply, it introduces a "good" gene into a person who has a disease caused by a "bad" gene.

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KidsHealth for Parents - Gene Therapy and Children