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Keim Lab Staff
Learn more about principal investigator Sarah Keim, PhD, MA, MS, and her dynamic team in the Keim Lab.
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Martin Lab Staff
Learn more about principal investigator Paul Martin, PhD, and his dynamic team in the Martin Lab.
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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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Surrogate Genes Help Shield the Muscle
While dystrophin and alpha-sarcoglycan gene replacement focus on replacing mutated genes, Muscle Group studies have also shown value in some existing “healthy” genes.
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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.
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Protein Acts as Antagonist to Strengthen Muscle
Another approach to improving the health of patients with neuromuscular diseases is to treat the life-threatening effects of the diseases, most notably the muscle degeneration that destroys patients’ ability to walk and ultimately takes their lives.
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Preventing Muscle Damage Caused by Inflammation
While myostatin blocks muscle differentiation and growth, inflammation contributes to muscle death in neuromuscular diseases. In muscular dystrophy, the immune process is heightened, which contributes to chronic inflammation that kills muscle cells.