Intellectual Property & Your Health Series - GMOs in medicine and research

Shidonna Raven
Jun 17
2 min read

Photo / Image Source: Unsplash,

Before you go to the Doctor!

GMOs in medicine and research

GMOs have emerged as one of the mainstays of biomedical research since the 1980s. For example, GM animal models of human genetic diseases enabled researchers to test novel therapies and to explore the roles of candidate risk factors and modifiers of disease outcome. GM microbes, plants, and animals also revolutionized the production of complex pharmaceuticals by enabling the generation of safer and cheaper vaccines and therapeutics. Pharmaceutical products range from recombinant hepatitis B vaccine produced by GM baker’s yeast to injectable insulin (for diabetics) produced in GM Escherichia coli bacteria and to factor VIII (for hemophiliacs) and tissue plasminogen activator (tPA, for heart attack or stroke patients), both of which are produced in GM mammalian cells grown in laboratory culture. Furthermore, GM plants that produce “edible vaccines” are under development. An edible vaccine is an antigenic protein that is produced in the consumable parts of a plant (e.g., fruit) and absorbed into the bloodstream when the parts are eaten. Once absorbed into the body, the protein stimulates the immune system to produce antibodies against the pathogen from which the antigen was derived. Such vaccines could offer a safe, inexpensive, and painless way to provide vaccines, particularly in less-developed regions of the world, where the limited availability of refrigeration and sterile needles has been problematic for some traditional vaccines. Novel DNA vaccines may be useful in the struggle to prevent diseases that have proved resistant to traditional vaccination approaches, including HIV/AIDS, tuberculosis, and cancer.

Genetic modification of insects has become an important area of research, especially in the struggle to prevent parasitic diseases. For example, GM mosquitoes have been developed that express a small protein called SM1, which blocks entry of the malaria parasite, Plasmodium, into the mosquito’s gut. This results in the disruption of the parasite’s life cycle and renders the mosquito malaria-resistant. Introduction of these GM mosquitoes into the wild could help reduce transmission of the malaria parasite. In another example, male Aedes aegypti mosquitoes engineered with a method known as the sterile insect technique transmit a gene to their offspring that causes the offspring to die before becoming sexually mature. In field trials in a Brazil suburb, A. aegypti populations declined by 95 percent following the sustained release of sterile GM males.

Finally, genetic modification of humans via gene therapy is becoming a treatment option for diseases ranging from rare metabolic disorders to cancer. Coupling stem cell technology with recombinant DNA methods allows stem cells derived from a patient to be modified in the laboratory to introduce a desired gene. For example, a normal beta-globin gene may be introduced into the DNA of bone marrow-derived hematopoietic stem cells from a patient with sickle cell anemia; introduction of these GM cells into the patient could cure the disease without the need for a matched donor.