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March 2012

Dr. Thomas Voit discusses gamma sarcoglycan gene therapy.

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July 2014

Dr. Scott Harper discusses RNAi silencing in limb girdle muscular dystrophy 1A.

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January 2013

Dr. James Ervasti discusses stability of therapeutic forms of dystrophin.

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February 2012

Dr. Burghes discusses antisense oligomer treatment in an SMA mouse model.

Article

June 2011

Dr. Paul Martin discusses how the CMAH gene deletion in the mdx mouse model of duchenne muscular dystrophy helps better mimic the more severe aspects of DMD.

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May 2012

Dr. Hugh Allen discusses cardiomyopathy in duchenne muscular dystrophy.

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News and Announcements

Stay up to date with the latest news coming out of the Office of Technology Commercialization.

Article

Amplifying Copy Numbers to Reduce SMA Severity

Spinal muscular atrophy (SMA) is a collection of different muscle diseases characterized by the selective loss of motor neurons in the spinal cord. Grouped together, SMA is the second leading cause of neuromuscular disease and is the leading genetic cause of death in infants and toddlers.

Article

Gene Therapy Going Strong

Gene Therapy Collaborative Group Targets Neuromuscular Disease At Nationwide Children’s Hospital, gene therapy strategies to reverse or prevent damage caused by muscle-wasting diseases are gaining strength, especially in animal models of human disease.

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Replacing Missing Genes

Duchenne muscular dystrophy (DMD) is the most common life-threatening childhood form of muscular dystrophy. It is characterized by progressive weakness and degeneration of skeletal muscles and caused by a mutation in the dystrophin gene.

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