Gene Therapy

About Gene Therapy

Gene therapy uses functioning genes as medicine for people who have a gene that is malfunctioning. Below is a breakdown of what genes are, how gene therapy works and why it’s being studied as a possible treatment for genetic disease now.

What are genes?

Genes have the information for making proteins. Which are responsible for doing most of the work in the body.  When genes don’t work properly, this may cause a genetic disease like beta-thalassemia.

  • Block 1

    Genes have the information for making proteins, which are responsible for doing most of the work in the body. The beta-globin gene contains the information for making the beta-globin protein. Beta-globin is a protein missing in beta-thalassemia.

  • Block 2

    The information in genes is contained in a molecule called DNA (deoxyribonucleic acid), which is found in almost every cell.

  • Block 3

    When a gene does not work properly, often caused by a mistake (known as a mutation) in the DNA, this may cause a genetic disease. This may result in an important protein, like  beta-globin, not being made.

HOW DOES GENE THERAPY WORK?

Gene therapy uses functioning genes as medicine for people who have a gene that is malfunctioning. The goal of gene therapy is to help correct the genetic disease by providing a functioning copy of the gene to make up for the malfunctioning gene.

Study participants are encouraged to talk their study team about gene therapy, chemotherapy and all the details associated with fertility and the options available to them at their study site. Fertility harvest, preservation and storage is available at no charge to people participating in any of the Northstar Studies. The options available may vary by study location.

WHY GENE THERAPY NOW?

Gene therapy has been studied as a potential treatment for genetic disease since the 1980s. Since then, significant advances have been made.

  • 30 years

    Gene therapy has been studied as a potential treatment for
    genetic disease2.

     

  • 2000

    Clinical trials have been conducted using different types of gene therapy in cancers, blood diseases, central nervous system disorders and immune system diseases2,3,5.

Sources:
  1. www.ornl.gov/sci/techresources/Human_Genome/medicine/ genetherapy.shtml (Accessed Dec. 4, 2012).
  2. Sheridan, C., 2011. Gene therapy finds its niche. Nature Biotechnology, 29(2), pp. 121–8.
  3. Kay, M. A., 2011. State-of-the-art gene-based therapies: the road ahead. Nature, 12(5), pp. 316–28.
  4. Moran, N., 2012. First Gene Therapy Approved. Nature Biotechnology. http://www.nature.com/nbt/journal/v30/n12/full/nbt1212-1153.html