Rapamycin allows gene therapy's results to improve
Used alone, gene therapy showed reduction in disease severity in the mice; however, used in combination with the drug rapamycin, it allowed all but a single mouse to regain their mobility functions. “Within a very short amount of time, we saw a nearly complete reversal of all the clinical signs and symptoms of the disease” said Hoffman. “Leading up to the project, I had spent nearly a decade studying the mechanisms of gene therapy-induced immune tolerance. Hence, I was confident that the gene immunotherapy would be effective, especially for preventing disease. However, my team and I were surprised and impressed at how effective it was at reversing pre-existing disease and restoring mobility in mice that were essentially paralyzed” he said.