Gene therapy is a new class of treatments that promise to treat and possibly cure many debilitating diseases. In contrast to conventional treatments, gene therapy treats the cause of the disorder rather than just the symptoms. Gene therapy functions by inserting new genetic material, either DNA or RNA, into a patient’s damaged cells. The most common way of inserting this new genetic material is by using a virus, a so called viral-vector. This new material inserted by the vector can then make up for the faulty DNA that was originally present. The choice of the vector that delivers this genetic material makes a large difference in how and where the new genes are integrated into the cell. Production of these vectors at scale is currently one of the major topics of research in gene therapy.
Patients suffering from the genetic condition haemophilia have a lack of coagulation factors that causes their blood not to clot. Specifically, factor VIII and IX for haemophilia A and B respectively. The result is frequent bleeding because their bodies are not able to stop the blood from coming out through the natural processes of clotting. This is potentially a life threatening situation if the bleeding occurs around the brain or other sensitive organs. The current treatment for haemophilia is regular injections of the missing clotting factors, in some cases, this can be as frequent as once every three days. Haemophilia results when someone only has a defective copy of the gene responsible for making these clotting factors making the afflicted unable to produce them in adequate amounts. The gene therapy approach to treating haemophilia is to insert a new functioning copy of the genes that encode for these clotting factors into the defective cells. If these new genes are integrated by the defective cells, then they will start to produce the clotting factors thus eliminating the need for regular injections. Instead of just treating the symptoms, lack of coagulation factors, gene therapy treats the true underlying problem of the bodies inability to produce the required amounts of the coagulation factors.
Author: Brian Ladd (ESR5)
