New Disease-Modeling Method: SMA in Mice (VIDEO)
Caption
Scientists have developed a new way of making animal models for human genetic diseases with pathology caused by errors in the splicing of RNA messages bearing protein-encoding instructions from genes. In this movie, mice modeling the devastating human motor-neuron illness spinal muscular atrophy (SMA) are shown with two healthy mice. The SMA mice are at an advanced stage of the illness; their mobility is severely impaired. Severe cases of the disease in humans can be lethal in the first years of life. The new modeling method provides a unique window on disease progression and a platform for the testing of novel therapeutics.
Credit
Adrian Krainer, Ph.D., Cold Spring Harbor Laboratory.
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