Rett syndrome…is a neurological developmental disorder and is classified as a particularly extreme form of autism. But, unlike most forms of autism, it almost exclusively shows up in girls.
[R]esearchers have been able to pinpoint the cause of the disorder — a mutation on the MeCP2 gene, carried on the X chromosome. And scientists at the Fred Hutchinson Cancer Research Center…[have reported] that they found a mechanism that could be used to treat Rett Syndrome, and possibly other diseases which are impacted by “silenced” genes — including cancer.
Here’s how a potential Rett Syndrome therapy would work: because female DNA has two X chromosomes, with the same genes on each, one is active and one is silenced in each cell. In girls that have Rett, only one of their X chromosomes carry that mutated gene, so about half of their active chromosomes carry it while half are normal.
“What we are trying to do here is to reactivate the silenced copy of the MeCP2 gene,” said Antonio Bedalov, a researcher at Fred Hutch who led the study.
[The study can be found here.]
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