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

Neuromuscular Disorders Program at Nationwide Children's

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.

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

First-Ever Patient-Initiated FDA Guidance To Accelerate Development and Review of Potental Therapies for Duchenne Muscular Dystrophy

Parent Project Muscular Dystrophy, an advocacy group founded by parents and family members of patients with Duchenne muscular dystrophy, recently submitted the first-ever patient-initiated guidance to the U.S. Food and Drug Administration for pharmaceutical companies to help expedite drug development for Duchenne. Kevin Flanigan, MD, principal investigator in the Center for Gene Therapy at The Research Institute, specializes in inherited muscular disorders and their potential therapies, and chaired one of seven working groups on the steering committee that drafted the guidance. 

Read the full story on Pediatrics Nationwide.

LivLife Makes Donation to Support MPS III Biomarker Research

LivLife Foundation recently donated $30,000 to Nationwide Children’s Hospital to support the collaborative MPS III biomarker research in the laboratories of Dr. Haiyan Fu and Dr. McCarty in the Center for Gene Therapy at The Research Institute. Dr. Fu said, “We truly appreciated the support from the MPS III community through LivLife. It comes at a critical moment. As we are moving our MPS IIIA and B gene therapy approaches forward towards clinical trials in patients, lack of biomarkers has become a challenge for the evaluation of therapeutic outcome.”

LivLife is a private foundation started by Mr. and Mrs. Jake and Kelly Hubert, in honor of their daughter Livia who suffers from MPS IIIA (Sanfilippo syndrome A), a devastating neurodegenerative lysosomal storage disease. LivLife has made great progress in raising public awareness about MPS III and raising funds to support MPS III research.

New Drug Demonstrates Improved Walking Performance over Placebo for Duchenne Muscular Dystrophy Patients

Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production of a protein vital to muscle growth and health. The study, led by a team in The Research Institute at Nationwide Children’s Hospital, is the first of its kind to show these results from an exon-skipping drug—a class of therapeutics that allows cells to skip over missing parts of the gene and produce protein naturally.

“I’ve been doing this for more than 40 years and this is one of the most exciting developments we’ve seen,” says Jerry Mendell, MD, lead author of the study and director of the Center for Gene Therapy at Nationwide Children’s. “It offers great hope to patients with Duchenne muscular dystrophy and their families.”

Read more.

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.

Read more

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.

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

Watch the video

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

Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000