X Chromosome Reactivation Provides A Potential Strategy For Treating X Linked Disorders
“Rett syndrome is a severe disorder in girls for which there currently is no available disease-specific treatment,” says lead author Lieselot Carrette, Ph.D., a research fellow in the laboratory of senior author Jeannie T. Lee, MD, Ph.D., MGH Department of Molecular Biology. “Targeting the ultimate cause of the disease would be expected to have better outcomes than addressing its many downstream effects.” Lee adds, “The approach described in our work takes advantage of the fact that every patient carries a cure within her own cells....