Using exon skipping technology in a model system of Ullrich congenital muscular dystrophy

From Muscular Dystrophy Campaign
A group of scientists from Italy has used exon skipping technology to restore the production of a protein called collagen VI in a model system of Ullrich congenital muscular dystrophy. The researchers used cells grown in the laboratory to demonstrate proof of principle that exon skipping might have the potential to be developed as a treatment for some people with this condition.
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  • Congenital Muscular Dystrophy

    A group of diseases causing muscle weakness at birth. Several defined genetic mutations cause muscles to break down faster than they can repair or grow. A child with CMD may have various neurological or physical impairments. Some children never gain the ability to walk, while others lose the ability as they grow older. Learn more...

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