Drug treats inherited form of intellectual disability in mice

Study suggests some symptoms in human version of the disorder could be eased.

Studying mice with a genetic change similar to what is found in Kabuki syndrome, an inherited condition of humans, Johns Hopkins researchers report they have used an anticancer drug to "open up" DNAi and improve mental function.

Along with a potential treatment for the intellectual disability seen in Kabuki syndrome, the study's findings also suggest a new way of thinking about a category of genetic conditions known as Mendelian disorders of the epigenetici machinery, the researchers say. In these disorders, a genetic mutation causes errors in the way proteins and chemicals bind to DNA, which in turn affects the rate at which DNA make proteins. In the case of a Kabuki syndrome-like condition in mice, the researchers found that those errors lead to a persistent but treatable decrease in new cell growth in one part of the brain. Their study adds to the growing evidence that intellectual disability may not always be irreversible.  Read more....

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