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SPOTLIGHT ON GENE THERAPY AND MUSCLE
MD Human Trials will Target Membrane
Article Illustration:
When dystrophin (dark blue) is missing or diminished, Duchenne or Becker MD results. Loss of any of the sarcoglycans (yellow) leads to a limb-girdle type of MD. When laminin-2 (green) is lost, a disorder results that's known as congenital MD. The small adeno-associated virus can be sued to deliver genes for the sarcoglycans, while the larger adenovirus will be needed to transport genes for dystrophin or laminin-2.
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