Genetically modified organisms (GMOs) are living entities whose genetic material has been altered through biotechnology to introduce, enhance, or remove specific traits. In medicine, this concept is used to create organisms that serve various healthcare purposes, from producing therapeutic substances to developing advanced treatments and diagnostic tools. The application of genetic modification in healthcare has opened pathways for innovations.
Producing Essential Medicines
Genetically modified organisms, primarily bacteria, yeast, or mammalian cell cultures, function as biological “factories” for producing therapeutic proteins and compounds. A landmark achievement was the production of human insulin, the first genetically engineered drug approved for use in 1982. Previously, insulin was sourced from animal pancreases, which presented issues with purity and supply.
Microorganisms like Escherichia coli bacteria are engineered to carry the human gene for insulin, enabling large-scale production. Beyond insulin, this technology yields human growth hormone, used to treat growth deficiencies, and clotting factors for individuals with hemophilia. Monoclonal antibodies, proteins designed to target specific cells or substances, are also produced using genetically modified mammalian cells. These engineered systems offer benefits such as high purity, abundant supply, and improved safety profiles compared to animal or human-derived substances.
Creating Advanced Vaccines
Genetic engineering has advanced vaccine development by enabling safer and more effective immunizations. This involves producing recombinant vaccines, where specific pathogen parts, such as a protein, are manufactured by genetically modified organisms. These components stimulate an immune response without introducing a weakened or inactivated whole pathogen, enhancing safety by eliminating the risk of causing disease.
Examples include the Hepatitis B vaccine, which uses genetically modified yeast cells to produce the Hepatitis B surface antigen. The Human Papillomavirus (HPV) vaccine also utilizes genetically engineered components to protect against the virus. These vaccines produce specific viral or bacterial proteins, leading to a more targeted and effective immune response.
Gene Therapy and Targeted Treatments
Genetic modification extends to direct medical interventions like gene therapy and gene editing, aiming to correct genetic defects or introduce new therapeutic functions within a patient’s cells. Genetically modified viruses or other vectors are frequently used as delivery systems to transport functional genes into target cells. These modified viruses are stripped of their disease-causing properties but retain their ability to efficiently deliver genetic material.
Gene editing technologies, such as CRISPR-Cas9, allow precise modifications to DNA directly within a patient’s cells. This technology can delete, insert, or modify specific genes, addressing the root cause of genetic disorders. Clinical applications are under investigation for conditions such as inherited blindness, sickle cell disease, and specific cancers. For instance, CRISPR-Cas9 has been explored to correct mutations for conditions like beta-thalassemia and to modify immune cells to better target cancer.
Innovations in Medical Diagnostics
Genetically modified organisms and their engineered components are instrumental in developing advanced diagnostic tools and tests. Genetic modification allows for the creation of organisms, such as bacteria, that can detect specific pathogens or disease biomarkers. These engineered biosensors convert the detection of a target molecule into a measurable signal, enabling rapid and sensitive diagnosis.
For example, genetically engineered bacteria have been developed to detect tumor DNA, offering a potential method for early cancer detection. These bacteria can be programmed to identify specific genetic mutations linked to cancer, providing a non-invasive diagnostic approach. Similarly, genetically engineered cells can produce highly specific antibodies used in diagnostic assays to identify disease markers or infectious agents. These applications enhance the accuracy, speed, and accessibility of disease detection, monitoring disease progression, and supporting medical research.