Bone marrow transplant stops symptoms of Rett syndrome in mouse model

Researchers from the Rett Syndrome Research Trust and the Rett Syndrome Research Trust UK have found that a bone marrow transplant can arrest some of the symptoms of the most physically disabling autism spectrum disorder in mouse models. Rett Syndrome causes loss of language skills, seizures, disordered breathing, tremors, and a range of other problems. It predominantly affects girls and symptoms usually appear between 6 and 18 months. The research team explored the function of microglia deficient in methyl-CpG binding protein 2 (Mecp2), the protein encoded by the ‘Rett gene.’ They found that when microglia lack properly functioning Mecp2, they cannot clean up cellular debris in the brain efficiently. Because microglia are derived from immune progenitor cells, it is possible to replace them via a bone marrow transplant. “Our encouraging results point to how surprisingly tractable this severe disorder proves to be, at least in the lab,” first author Noël C. Derecki said.