Gene therapy research on mice

Gene therapy research on mice

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Caption: Gene therapy research on mice. This mouse is a mammalian model for the disorder Duchenne Muscular Dystrophy (DMD), a condition in humans typified by muscle wasting and loss of function. The mouse has an abnormal gene that, left untreated, caused the muscles in its legs to waste, making it unable to walk. Here, it has been treated by a method of gene therapy known as exon skipping. It is undergoing tests to determine how much its muscles have recovered after treatment. Photographed at the AFM (French Muscular Dystrophy Association), Evry, France.

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