Center for Gene Therapy :: The Research Institute at Nationwide Children's Hospital

Clinical Trials at The Center for Gene Therapy

Center for Gene Therapy Faculty

 
Affiliate Faculty

Neuromuscular Disorders Program at Nationwide Children's

The multidisciplinary Neuromuscular Disorders Program at Nationwide Children's diagnoses and treats disorders affecting peripheral nerves and muscles in children, such as muscular dystrophy, myositis and congenital myopathy.

Visit the Neuromuscular Disorders Program page

Visit the Neurosciences Center pages

Gene Therapy

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.

 

CENTER HIGHLIGHTS

Wellstone Muscular Dystrophy Cooperative Research Center

The National Institutes of Health has designated the Center for Gene Therapy as a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC).  MDCRCs promote basic, translational and clinical research and provide important resources that can be shared within the national muscle biology and neuromuscular research communities.

The MDCRC will allow Nationwide Children's researchers to further develop methods to overcome immune barriers to gene correction for Duchenne muscular dystrophy.

GMP/Cleanroom Facility

The Center for Gene Therapy and the Viral Vector Core are home to a Good Manufacturing Practice (GMP) production facility for manufacture of clinical-grade rAAV vectors.

Clinical Trials

Investigators with the Center for Gene Therapy currently are conducting numerous clinical research studies, especially for neuromuscular disorders.

OSU/Nationwide Children's Muscle Group

The OSU and Nationwide Children's Muscle Group brings together investigators with diverse research interests in skeletal muscle, cardiac muscle, and neuromuscular biology.

Learn how the 24 labs within OSU/Nationwide Children's Muscle Group are working to improve approaches to treat muscle injury and disease. Read about how their collaborations are changing the way we treat neuromuscular diseases.

This Month in Muscular Dystrophy Podcast

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.

CENTER NEWS

Scientists Studying Rare Diseases Quoted in Wall Street Journal Article

Center for Gene Therapy investigators Doug McCarty, PhD, and Kevin Flanigan, MD, were recently quoted in a Wall Street Journal article, "Families Push for New Ways to Research Rare Diseases." Our researchers will soon launch a study related to the rare disorder, Sanfilippo Syndrome, a disease where the child is missing or has insufficient amounts of one of four enzymes needed to break down sugar molecules. Children with Sanfilippo Syndrome will ultimately lose their ability to walk, talk and eat.

Read the article here.

Exon-Skipping Shows Promise in Duchenne Muscular Dystrophy

Results from a Phase IIb extension trial of the drug eteplirsen show an increased ability to walk in boys with Duchenne muscular dystrophy.

Read more

Simplified Newborn Screening for Duchenne Muscular Dystrophy Shows Promise as an International Model 

Investigators in the Center for Gene Therapy have developed an approach to newborn screening for the life-threatening genetic disorder, Duchenne muscular dystrophy (DMD), and potentially other muscular dystrophies.  

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New Model System Offers Clues to ALS

Using tissue samples collected from patients with amyotrophic lateral sclerosis, scientists have created a new in vitro model for the disease that is providing insights into the mechanism of the disorder.  Findings appear in Nature Biotechnology.

Access the study abstract

Access the JAMA commentary

Access a summary of this study

NIH Grant Helps Move Toward Clinical Trials Targeting the Lysosomal Storage Disease MPSIIIB

To date, the greatest challenge in developing therapies for MPS IIIB has been the presence of the blood-brain barrier, which prevents therapeutics from entering the central nervous.  New funding will allow investigators complete necessary preclinical studies and to submit an investigational new drug application to the FDA for a Phase I/II AAV9 gene therapy clinical trial in patients with MPS IIIB.

Read more :: NIH Grant Helps Move Toward Clinical Trials Targeting the Lysosomal Storage Disease MPSIIIB

Gene Therapy Reveals Unexpected Immunity to Dystrophin in Patients with Duchenne Muscular Dystrophy

An immune reaction to dystrophin, the muscle protein that is defective in patients with Duchenne muscular dystrophy, may pose a new challenge to strengthening muscles of patients with this disease, suggests a new study appearing in the October 7, 2010, issue of The New England Journal of Medicine.  This research is part of the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center at Nationwide Children's Hospital.

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

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

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