Gene therapy: FAQ

Written by RegMedNet

As part of our gene therapy spotlight, we have answered the most frequently asked questions relating to gene therapy.

What is gene therapy?

Gene therapy is an emerging field that involves inserting genetic material into patient tissues in an attempt to prevent, alleviate or cure a condition. The genetic material inserted during gene therapy can either provide the cell with new activities, restore mechanisms broken by mutations, or counteract faulty activities the cell has acquired.

What can gene therapy treat?

In theory, gene therapy could be used to treat any condition which is the result of a mutation or cellular irregularity; however, due to current limitations, the conditions that are focused on tend to be “Mendelian” conditions with simple genetic causes, rather than complex genetic conditions that are the result of multiple mutations. Key conditions that have been intensely tested include cystic fibrosis and Duchenne muscular dystrophy.

How does gene therapy work?

Gene therapy utilizes a vector, such as a manipulated viruses or liposome, to administer genetic material to a patient or a collection of cells. As the DNA enters its target it can compensate for the failure of the diseased cells by producing the same product or a synthetically designed one.

Gene therapy can be performed either directly on patients or in collaboration with cell therapy, where cells are treated within the lab and then transferred into the patient to correct the condition.

Can gene therapy work with stem cells?

Stem cells often seem resistant to the vectors used in gene therapy; however, it is possible to perform gene therapy upon them. Modifying stem cells has been a major hinderance to successful gene therapy being utilized within the clinic, as most therapies would require stem cells to be modified to allow for permanent correction within the tissues.

Can gene therapy cure cancer?

Whilst not widely utilized, it has been suggested that gene therapy could treat cancer. The idea focuses on manipulating the cancerous tissues and introducing cellular sensitivities or a lethal gene; however, the idea has yet to gain traction and move to the clinic. One of the major hurdles for the treatment is selecting only cancerous cells without targeting the healthy cells and ensuring complete eradication of the cancer. Although gene therapy on its own has gained little traction, CAR-T therapy has become increasingly popular. The procedure — a combination of cell therapy, gene therapy and immunology — involves the insertion of genetic material into T-cells to modify their behavior, allowing them to target cancerous cells within a patient.

Read more about gene therapy in our spotlight.