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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.
Article
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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.
Article
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.