Gene therapy research on mice. Confocal light micrograph of a mouse forelimb before (left) and after (right) gene therapy. The 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 reduces dystrophin production, which causes muscle loss. Dystrophin is a protein important for maintaining normal muscle function. The necrotic muscle fibres prior to treatment are orange and yellow. Dystrophin is green. When the mouse was treated by a method of gene therapy known as exon skipping, it regained normal dystrophin levels and muscle function, as shown at right.
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