Intravenous Gene Therapy Protects Normal Tissue of Mice During Whole-Body Radiation
News Nov 07, 2007
Gene therapy administered intravenously could be an effective agent to protect vital organs and tissues from the effects of ionizing radiation in the event of large-scale exposure from a radiological or nuclear bomb, according to an animal study presented by University of Pittsburgh researchers at the 49th annual meeting of the American Society for Therapeutic Radiology and Oncology (ASTRO) in Los Angeles.
“Ionizing radiation can be extremely damaging to cells, tissues, organs and organ systems,” said Joel S. Greenberger, M.D., professor and chairman, department of radiation oncology, University of Pittsburgh School of Medicine.
“In previous studies, we demonstrated that gene therapy can be both swallowed in liquid form and inhaled through a nebulizer prior to radiation exposure to protect healthy tissues from damage. In this study, we found that the same therapy administered intravenously also offers protection during exposure to whole-body irradiation.”
Dr. Greenberger added that intravenous administration could potentially offer wide-reaching protection to the public in the event of a terrorist attack since experts believe a significant number of the population would die within 30 days of receiving a large dose of radiation to the entire body.
In the study, mice were used to test the protective effects of manganese superoxide dismutase plasmid liposome (MnSOD-PL) gene therapy on the bone marrow during whole-body irradiation.
The researchers found that in a control group of mice that received an initial 9.5 Gy dose of radiation, 58 percent survived at 30 days compared to 90 percent after the same length of time for an experimental group of mice that were injected with MnSOD-PL prior to irradiation. Between 30 and 330 days, there were no differences in survival rates between experiment and control group mice, indicating that systemic MnSOD-PL treatment was not harmful to survival.
“Intravenous administration of gene therapy appears to prevent the damaging effects of radiation, suggesting it is a viable delivery method,” said Dr. Greenberger. “Future clinical studies will tell us whether this therapy can protect people from the deadly effects of radiation.”
As genome editing technologies advance toward clinical therapies, they are raising hopes of a completely new way to treat disease. However, challenges need to be addressed before potential treatments can be widely used in patients. To tackle these challenges, the National Institutes of Health has launched the Somatic Cell Genome Editing program, which has awarded multiple grants including more than $3.6 million to assess the safety of genome editing in human cells and tissues.