Nationwide Children's News Releases

Mar 19, 2012

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

Investigators at Nationwide Children’s Hospital, working with the DNA Sequencing Core Facility at the University of Utah, have developed an approach to newborn screening (NBS) for the life-threatening genetic disorder, Duchenne muscular dystrophy (DMD) and potentially other muscular

Feb 29, 2012

Nationwide Childrens Hospital Neuromuscular Disorder Podcasts Now Available on iTunes

In 2010, the Center for Gene Therapy at Nationwide Children’s Hospital launched a monthly podcast entitled, “This Month in Muscular Dystrophy,” featuring internationally known scientists discussing the latest research in muscular dystrophy and other neuromuscular disorders.

Feb 22, 2012

Study: Muscle Regeneration May Provide Ideal Environment for Rhabdomyosarcoma

Inflammation, cell division and cell differentiation that occur during skeletal muscle regeneration may provide an ideal environment for the highly malignant tumor, rhabdomyosarcoma to arise.

Feb 15, 2012

Grant Provides Funding Toward Gene Therapy Clinical Trial to Replace the DMD Gene in All of the Leg Muscles of Duchenne Patients

Investigators in the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital are working toward an approach to replace the defective gene in Duchene muscular dystrophy (DMD) by treating all of the muscles in the leg, thanks to funding from Jesse’s Journey.

Nov 02, 2011

Nationwide Children's Hospital and Ohio State Researchers Design a Viral Vector to Treat a Genetic Form of Blindness

Researchers at Ohio State University Medical Center and Nationwide Children’s Hospital have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited, progressive form of blindness that affects mainly males.

Oct 03, 2011

Nationwide Childrens Hospital Researchers Receive Neurology Award for Contribution to Clinical Neuroscience

Jerry Mendell, MD, director of the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital, and his fellow researchers, are the latest recipients of the Annals of Neurology prize for an outstanding contribution to clinical neuroscience. The study which helped Dr.

Aug 10, 2011

Human-Cell-Derived Model of ALS Provides a New Way to Study the Majority of Cases

For decades, scientists have studied a laboratory mouse model that develops signs of the paralyzing disease amyotrophic lateral sclerosis (ALS) as they age.

May 25, 2011

Nationwide Childrens Hospital Researchers Receive NIH Grant for the Move Toward Clinical Trials Targeting the Lysosomal Storage Disease MPSIIIB

Investigators at Nationwide Children’s have received a grant from the National Institutes of Health (NIH) to help move a therapy for MPS IIIB that has been shown effective in mice toward clinical trials in humans.

Jan 10, 2011

Patient-Derived Embryonic Stem Cells Help Deliver Good Genes in a Model of Inherited Blood Disorder

Researchers at Nationwide Children’s Hospital report a gene therapy strategy that improves the condition of a mouse model of an inherited blood disorder, Beta Thalassemia. The gene correction involves using unfertilized eggs from afflicted mice to produce a batch of embryonic stem cell lines.

Oct 06, 2010

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.