Patient Education & Support
Human DNA is approximately 3 billion base pairs long and it contains about 30,000 genes. We typically get two copies of each gene from our parents. They influence everything from our physical traits to physiological properties including our immune system. However, genes don’t always work to our advantage, or sometimes they are not built correctly. Every protein, enzyme, functional and signaling molecules we make in our cells are made from their templates in our DNA. A small alteration in our DNA results in the formation of a different, sometimes defective, structure. We may be born with such defects, genes can change as we age, or they can be altered or damaged by external forces like chemicals and radiation.
When a gene mutates -or changes- in a way that causes disease, gene therapy may be able to help. Gene therapy is the therapeutic delivery, removal or editing of the genetic material in a patient’s cell to treat a disease. Gene therapy has been a main research field in medicine for over 40 years. In gene therapy, the genetic material is delivered into the cell by using a vehicle called a “vector”. Typically, certain artificial viruses are used as vectors because of their ability to stealthily enter into a cell and affect its genes. The vector can either be delivered into a cell outside the body (ex-vivo) or the vectors can be directly injected into the body (in-vivo).