Search Results
Viewing: 1301-1310 of 11587 | All
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.
Article
Circulating "Good" Genes Body-Wide
While Muscle Group members are discovering new targets for drug development, progress will continue to be stunted if the drug can’t reach the central nervous system, a primary player in neuromuscular diseases.
Article
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.
Article
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.
Article
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.
Article
Frequently Asked Questions
Find answers to the most common questions around technology commercialization.
Article
Startups
Learn more about our startups that began with research and innovation at Nationwide Children’s.
Article
Transfer to Transform 2025
Transfer to Transform, published annually online and in print, shares the story of Technology Commercialization at Nationwide Children's — a story of bold thinkers, exciting new technologies and job-producing startups.