Researchers at the Board of Governors Gene Therapeutics Research Institute at Cedars-Sinai Medical Center have shown for the first time that it is possible to sustain therapeutic gene expression in the central nervous system for up to a year, even in the presence of an anti-viral immune response mechanism that is normally present in humans.
The researchers demonstrated in an animal model that the delivery system for the gene, a novel gutted adenoviral vector called HC-Adv, is completely invisible to the immune system. Vectors previously used to deliver genes carried minute amounts of viral proteins that were detected by the immune system, triggering an immune response that rendered the therapeutic gene inactive after a period of weeks.
According to the researchers, this delivery system is safer and more effective than what is currently available, and should therefore advance clinical gene therapy trials for people suffering from central nervous system disorders such as Parkinson's, Alzheimer's, and Multiple Sclerosis. The research was sponsored in part by The National Institutes of Health.
The full study can be accessed at www.moleculartherapy.org
Pedro R Lowenstein, M.D., Ph.D., Director of the Board of Governors Gene Therapeutic Research Institute, Cedars-Sinai Medical Center is principal investigator of this study and is available for interviews.
The Board of Governors Gene Therapeutic Research Institute at Cedars-Sinai Medical Center is a world-renowned translational research program. Established in 2000, the institute is engaged in state-of-the-art technologies to develop genetic and stem cell-based therapies for the treatment of life-threatening disorders such as cancers, chronic neurodegenerative diseases and autoimmune disorders. It includes more than 30 scientists and physicians devoted to bringing these new therapeutic approaches to the clinical arena. Additional information is available at www.cedars-sinai.edu/gtri.
*Study appeared online in Molecular Therapy, the official journal of The American Society of Gene Therapy