Clinical Trials at The Center for Gene Therapy
GMP Facility
Center for Gene Therapy Faculty
OSU/Nationwide Children's Muscle Group
The mission of the Center for Gene Therapy is to investigate and employ the use of gene and cell based therapeutics for prevention and treatment of human diseases including: neuromuscular and neurodegenerative diseases, lysosomal storage disorders, ischemia and re-perfusion injury, neonatal hypertension, cancer and infectious diseases.
Learn about our areas of focus and featured research.
$7 Million NIH Grant Designates Nationwide Children’s as a Wellstone Center to Advance Muscular Dystrophy Research
A $5 million grant from the National Institutes of Health (NIH) will help researchers at Nationwide Children’s Hospital translate new scientific findings and technological developments into novel treatments for the muscular dystrophies. The grant designates Nationwide Children’s Hospital as a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, one of three national award recipients in 2010.
Read more :: Nationwide Children's Designated as Wellstone Center
Nationwide Children’s and OSU Unite to Target Muscle Regeneration in Degenerative Diseases
In total, 24 labs are working to improve approaches to treat muscle injury and disease. Read about how their collaborations are changing the way we treat neuromuscular diseases.
- Gene Therapy Going Strong
- Replacing Missing Genes
- Surrogate Genes Help Shield the Muscle
- Amplifying Copy Numbers to Reduce SMA Severity
- Protein Acts as Antagonist to Strengthen Muscle
- Preventing Muscle Damage Caused by Inflammation
- Circulating “Good” Genes Body-Wide
- Early Detection and Personalizing Treatments
Listen Now: Podcasts Dedicated to the Latest Neuromuscular Research
Hosted by Kevin Flanigan, MD, "This Month in Muscular Dystrophy" podcasts highlight the latest in muscular dystrophy and other inherited neuromuscular disease research. During each podcast, authors of recent publications discuss how their work improves our understanding of inherited neuromuscular diseases, and what their work might mean for treatment of these diseases.
Gene Therapy News
Spinal Muscular Atrophy May Also Affect the Heart
Along with skeletal muscles, it may be important to monitor heart function in patients with spinal muscular atrophy (SMA). These are the findings from a study conducted by Nationwide Children’s Hospital and published online ahead of print in Human Molecular Genetics. This is the first study to report cardiac dysfunction in mouse models of SMA.
Read more :: Spinal Muscular Atrophy May Also Affect the Heart
Parent Project Muscular Dystrophy Awards $600K to Nationwide Children’s Hospital for Gene Therapy Study
Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced that PPMD will award a $600,000 grant to Nationwide Children’s Hospital in Columbus, Ohio to conduct clinical testing of a promising gene therapy technique for muscle disease.
Read more :: Parent Project Muscular Dystrophy Awards $600K to Nationwide Children’s Hospital for Gene Therapy Study
New Mouse Model of DMD More Accurately Mimics Human Disease
Therapies to treat Duchenne muscular dystrophy may sooner progress toward clinical trials thanks to a more realistic mouse model that mimics the human disease better than existing models. The new framework is detailed in a Nationwide Children’s Hospital study that appears in Science Translational Medicine.
Read more : New Mouse Model of DMD More Accurately Mimics Human Disease
Study Examines Gentamicin Treatment of DMD
A recent Center for Gene Therapy study appearing in the Annals of Neurology discusses the therapeutic concept of mutation suppression, even though clear clinical efficacy was not achieved.
Read the study: Gentamicin treatment of DMD
New Gene Therapy Study Reverses Effects of Lethal Childhood Muscle Disorder in Mice
Reversing a protein deficiency through gene therapy can correct motor function, restore nerve signals and improve survival in mice that serve as a model for the lethal childhood disorder spinal muscular atrophy, new research shows.
Read more about: New Gene Therapy Study Reverses Effects of Lethal Childhood Muscle Disorder in Mice.
Investigators Identify Gene Mutations that Predispose Patients with Becker Muscular Dystrophy to Early Onset Cardiomyopathy
Investigators in The Research Institute at Nationwide Children’s Hospital have identified a link between specific modifications of the dystrophin gene and the age of cardiac disease onset in patients with Becker muscular dystrophy.
Read more about the gene mutation that predisposes patients with BMD to early onset cardiomyopathy
Cancer/Gene Therapy Study Ranked as Top 10 Key Research in 2009
A study conducted by Jerry Mendell, MD, Janiah Kota, PhD, and colleagues in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, in collaboration with Johns Hopkins University, has been designated as one of Nature Medicine’s top 10 key papers published in 2009. Nature Medicine is a premier journal for biomedical research.
The paper details a promising new cancer-stopping therapy. The researchers discovered that delivering microRNAs, small molecules that are highly expressed in normal tissues but lost in diseased cells, can result in tumor suppression.
Read more about the promising new cancer-stopping therapy
Mendell Designated 2009 University Distinguished Scholar
Jerry R. Mendell, MD, has received the 2009 University Distinguished Scholar Award from the Department of Research at The Ohio State University.
The Distinguished Scholar Award, established in 1978, recognizes exceptional scholarly accomplishments by senior professors who have compiled a substantial body of research. Distinguished Scholars receive a $3,000 honorarium and a research grant of $20,000 to be used over the next three years.
FSH Society Fellowship Funds Facioscapulohumeral Muscular Dystrophy Research
Utilizing this one-year, $40,000 fellowship grant, Dr. Harper and his team will investigate the role of the DUX4 gene in FSHD pathogenesis. Read more :: FSH Society Fellowship Funds Facioscapulohumeral Muscular Dystrophy Research >>
