A collaborative effort between three scientists at UMass Chan Medical School is expected to help advance genome editing approaches for Rett syndrome, a neurological disease caused by mutations in a gene called MECP2, which is a master regulator of gene expression in the brain.
Erik Sontheimer, PhD, the Pillar Chair in Biomedical Research and professor of RNA therapeutics; Jonathan Watts, PhD, professor of RNA therapeutics; and Scot Wolfe, PhD, professor of molecular, cell & cancer biology, were awarded $2.3 million in new funding from the Rett Syndrome Research Trust to utilize base-editing and prime-editing technologies to correct mutations in MECP2.
“We are working on different aspects of genome editing systems for correction of mutations in the genome. For several years now, we’ve been working together on developing tools for correcting common mutations in Rett syndrome and then working to apply those tools using delivery systems to the central nervous system. This funding helps to continue to support those efforts,” Dr. Wolfe said.
