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.
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.
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.
The information in genes is contained in a molecule called DNA (deoxyribonucleic acid), which is found in almost every cell.
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.
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.
Gene therapy has been studied as a potential treatment for genetic disease since the 1980s. Since then, significant advances have been made.