Yale researchers find potential treatment for Rett Syndrome |
Yale researchers have found an experimental cancer drug that can extend life in Rett Syndrome in animal studies.Rett Syndrome is a devastating genetic disorder that afflicts about one of every 10,000 to 15,000 girls within 6 to 18 months after birth.The findings of the research have been published in the journal Molecular Cell.
Rett Syndrome is a rare brain condition related to autism, caused by mutations in the MECP2 gene. Because the gene is located on the X chromosome, the syndrome occurs almost exclusively in girls. No drugs are available to treat Rett.
The road to a cure for Rett syndrome is paved with encouraging findings but also with many challenges. Whilst optimism drives discovery, it is important for those directly affected to be well informed.
In addition, the drug JQ1 also restores the cellular function of neurons in human models of the disease. Rett Syndrome causes severe deficits in language, learning and other brain functions and eventually leads to death, often during teenage years.
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