Precision epigenome editing can repair genetic syndrome of intellectual disability

Precision epigenome editing can repair genetic syndrome of intellectual disability

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Dec 13 2019

Using a targeted gene epigenome editing approach in the developing mouse brain, Johns Hopkins Medicine researchers reversed one gene mutation that leads to the genetic disorder WAGR syndrome, which causes intellectual disability and obesity in people. This specific editing was unique in that it changed the epigenome — how the genes are regulated — without changing the actual genetic code of the gene being regulated.