Viruses and bacteria can be a devastating cause of disease, but bioengineers, virologists, and microbiologists in the Icahn Genomics Institute (IGI) and elsewhere are working to harness these organisms to create powerful new medical treatments for a wide variety of diseases. Through extensive investigation, scientists have removed harmful genes from adenoviruses, adeno-associated virus (AAV), retroviruses, lentiviruses, and other viruses and turned them into vehicles for delivering synthetic genes into cells to reprogram cellular functions or to replace defective genes. This has led to highly effective treatments for congenital blindness, immunodeficiencies, bleeding disorders, spinal muscular atrophy, and cancer.
Viruses and virus derivatives can also be used to create vaccines to prevent infectious diseases or as powerful immunotherapy treatments that kill cancer cells and recruit the immune system to eliminate tumors. Work by Dr. Adolfo Garcia-Sastre and others at Mount Sinai have turned New Castle Disease Virus (NDV) into an effective new COVID-19 vaccine as well as an anti-cancer agent, harnessing NDV’s potent ability to turn on a type I interferon response and stimulate the immune system.
Commensal bacteria—the bacteria that live in our gut, our mouths, and on our skin—are also being used to treat disease. Studies at Mount Sinai have found that a person’s unique microbiome composition can have a profound impact on health, including associations with asthma and food allergy. Scientists in the IGI have generated one of the largest biobanks of human gut bacteria in the world and are using it to identify the specific microbes that affect inflammation and metabolism, and promote different diseases, including inflammatory bowel disease (IBD). They are also using these biobanks to manufacture microbiome cocktails that will undergo clinical trials to assess their ability to improve patient outcomes in IBD and other inflammatory diseases as well as the immunotherapy response in cancer.