Mark Zylka discovered that a class of drugs called topoisomerase inhibitors reduces the expression of long genes, raising the possibility that this class of drugs could be clinically relevant for Rett. One such drug is topotecan which is FDA approved for cancer. While the Greenberg lab at Harvard Medical School is currently testing Topotecan in Rett mice models, the drug may not be ideal since it doesn't get into the brain easily and would be toxic for long term use. As a result, Dr. Zylka has been awarded $400,000 from RSRT to screen for other compounds that can rebalance expression of long genes safely.
For more information, visit the RSRT website.