What is gene therapy?

Written by RegMedNet

As part of our spotlight on gene therapy, we’ve rounded up answers to some frequently asked questions. Got one you’d like an answer to? Leave a comment below!

What is gene therapy?

In a gene therapy, genes are introduced into a cell that which correct or replace defective genes, which might be causing a disease or disorder.

How does gene therapy work?

Gene therapy can take place ex vivo, outside the body, or in vivo, inside the body. In an ex vivo therapy, genes are delivered to a sample of cells taken from the body using a vector, such as a virus, then infused back in. In an in vivo gene therapy, the replacement or corrective genes are delivered directly to where they are needed to act on a particular group of cells.

What can gene therapy treat?

Gene therapy could treat a range of diseases, including hereditary genetic diseases, such as sickle cell anemia. This represents an unmet need, as often, only symptoms of these diseases can be treated, as opposed to the disease itself.

Why is gene therapy beneficial?

Gene therapy has many potential benefits over traditional pharmaceuticals. According to Mohammed Asmal, bluebird bio (MD USA), because gene therapy involves an autologous transplant, “there is no risk of GvHD or graft rejection”. Gene therapies also offer the potential for “curative treatment”, says Nafees Malik, Asklepian Consulting (London, UK), so they could be a one-time treatment. This could save money in patient care in the long-term.

Gene therapy vs cell therapy?

Each kind of therapy has its own risks and benefits. Both offer the potential for a curative therapy and are best suited to different kind of diseases. For example, cell therapy can be used to treat diseases where there are defective or a deficit of cells, for example in spinal cord injuries or heart conditions. However, cell therapy offers a small risk of tumorigenicity and gene therapy could lead to off-target effects where the treatment leads to harmful, additional mutations.

Learn more about gene therapy in our latest spotlight>>