• What is Gene Therapy?

    Gene Therapy for Children

    Phone: 617-632-5064

    Gene therapy is a technique through which genes are added or replaced to treat or prevent disease.

    Our genes, which hold the code for all of our body's functions, are made of DNA. Damage to DNA, such as a mutation, is an underlying cause of the genetic defects that lead to cancers, blood disorders, and other conditions. Gene therapy delivers DNA into a patient’s cells to replace faulty or missing genes – or add new genes – in an attempt to cure cancer or make changes so the body is better able to fight off disease.

    Scientists are investigating a number of different ways to do this:

    • Replacing missing or mutated genes, which is the most common approach
    • Changing the regulation of genes; mutated genes that cause cancer could be turned off so they don’t cause disease or turned on to fight disease.
    • Making cancer cells more recognizable to the body’s immune system to improve the body’s disease-fighting response, also known as immunotherapy.

    How does gene therapy deliver new genes into cells?

    With gene therapy, the DNA for the new or corrected gene or genes is carried into a patient’s cells by a delivery vehicle called a vector, typically a specially engineered virus. The vector then inserts the gene(s) into the cells' DNA.

    What are the steps of gene therapy?

    For patients, the process for delivering genes to cells is fairly simple. Check out this image of the gene therapy process:

    Basic Process of Gene Therapy

    Learn more: