• Menkes Disease Treatment on the Horizon, After Nearly Three Decades

    15 days ago - By DNA Science

    Headlines often trumpet the latest in gene editing, RNA drugs, or gene therapy. The less buzzy, but more classic strategy of providing a nutrient that a genetic glitch blocks, has been quietly making strides against Menkes disease, which impairs copper absorption. November is Menkes disease awareness month.
    Copper Deficiency
    Menkes disease results from a mutation in a gene on the X chromosome, so its affects boys. About 70% inherit the mutation from their mothers, who are carriers. The rest have a new mutation that arises in egg or sperm.
    The healthy version of the gene encodes a protein...
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