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.
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“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.]The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: Fred Hutch researchers discover way to re-activate ‘silenced’ genes, in possible treatment for Rett Syndrome and other diseases
