All the body’s components are genetically represented in the human genome. Each disease, whether it is genetic, infectious, or environmentally caused involves a genetic component. The ability to alleviate or cure diseases through the genetic axis has tremendous potential. Potential cures for hemophilia, AIDS, and various forms of cancer are in progress.
In hemophilia A, lentiviral vectors could be used to express the Factor VIII protein by engineering the body’s hematopoetic stem cells. The gene would be delivered into stem cells using a lentiviral vector that efficiently delivers payloads to these cells. Other diseases, particularly blood related, could be effectively targeted by lentiviral vectors that express specific payload genes to alleviate various diseases. This could be achieved by either regenerating various cells and tissues or restoring endogenous production of a particular biochemical.
Although current drug therapies can effectively manage AIDS, they do not represent a cure. The problem with long-term management of AIDS with antiretroviral drug therapy is the cumulative toxicities endured with prolonged drug use. In addition, viral resistance is making these therapies increasingly ineffective. A lentiviral vector armed with an anti-HIV payload gene could be delivered into the cells that HIV intends to infect, making them resistant to infection.
The efficient delivery properties of lentiviral vectors could also be used to deliver genes in a variety of anticancer strategies, including stem cell therapy. One method is to improve existing therapies by limiting their toxicity. Lentiviral vectors are now being used to deliver detoxifying genes to make these cells resistant to the toxic effects of chemotherapy.