2008 Notable Research Accomplishments
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2008 Notable Research Accomplishments

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The Research Institute at Nationwide Children's Hospital notable research accomplishments for 2008, taken from the Annual Report

The Research Institute at Nationwide Children's Hospital notable research accomplishments for 2008, taken from the Annual Report

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2008 Notable Research Accomplishments 2008 Notable Research Accomplishments Document Transcript

  • Notable Research Accomplishments O f the more Dr. Brian Kaspar and his colleagues in than five the Center for Gene hundred Therapy reported an important step forward scholarly in their ongoing work to test gene therapeutic works approaches for muscular published dystrophy. Muscular dystrophy, often caused by faculty at Nationwide Children’s by a mutation in the dystrophin gene, is a serious Hospital in 2008, the following inherited disorder in which muscles become progressively weak, damaged and wasted. There publications are highlighted for a variety is no cure and life expectancy is dramatically reduced. An obvious therapeutic tactic to treat of reasons. For example, some report this devastating disease would be to use gene seminal contributions to the field and therapy to replace the defective dystrophin gene. are published in leading biomedical While this approach is being developed in the Center for Gene Therapy, there are complex journals. Others demonstrate the unique technical challenges unique to the dystrophin capabilities of scientists at Nationwide gene. Dr. Kaspar conceived of a less complex molecular approach that involves myostatin, a Children’s Hospital or emphasize the human protein that naturally acts as a brake on muscle growth. He reasoned that long-term outstanding clinical expertise of our inhibition of myostatin using gene therapy might faculty. Throughout this report are increase muscle mass. The investigative team used a single dose of intramuscular adeno-associated catalogued hundreds of other virus vector housing the myostatin-inhibitor scientific contributions by our faculty follistatin to assess effects on muscle mass and function. Significantly increased muscle mass and to the understanding of pediatric health strength was found in experimental models of muscular dystrophy. This study advanced the and disease. As a leading pediatric group’s progress in designing novel molecular biomedical research institution, approaches for treating muscular dystrophy in children. The Center for Gene Therapy is a Nationwide Children’s Hospital is a sterling example of a translational research group, major contributor to new knowledge with team members capable of carrying experimental approaches from the laboratory that will lead to improvements in the bench to the patient’s bedside. lives of both ill and well children. – Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors. Proceedings of the National Academy of Sciences, USA. 2008 Mar 18;105(11):4318-22. The Research Institute at Nationwide Children’s Hospital :: P A G E 3
  • Christy Collins and outflow tract (LVOT) anomalies in 91 Dr. Dawn Comstock, children. This category of congenital heart investigators in the defects includes mild lesions like bicuspid aortic Center for Injury valves, and far more serious defects like congenital Research and Policy, aortic stenosis, aortic coarctation and hypoplastic studied the epidemi- left heart syndrome. The latter disorder is fatal ology of injuries in without urgent intervention in the newborn high school baseball period. Dr. McBride found mutations in a players. A novel and major developmental regulatory protein called innovative feature NOTCH in 15.4% of children with LVOT, of this research is the use of Reporting Injuries versus 3.9% of controls. These mutations Online (RIO), a national web-based high school affected NOTCH signaling in in vitro assays, injury reporting tool recently invented by Dr. proving the biological significance of the Comstock and collaborators at The Research findings. This important study adds further Institute. The power of RIO is that large support for a genetic origin for many cases of numbers of injuries from all over the United congenital heart disease and a hope for early States can be recorded by coaches and trainers detection, fetal therapy or even prevention. into the web-based system and then analyzed by – NOTCH1 mutations in individuals with left Dr. Comstock and her team. Using RIO, details ventricular outflow tract malformations reduce ligand- of 131,555 high school baseball injuries were induced signaling. Human Molecular Genetics. 2008 Sep analyzed in detail. Distinctive findings emerged. 5;17(18):2886-93. For example, head/face and mouth/teeth injuries Dr. Jeff Bridge and for pitchers, infielders and batters were far more his colleagues in the common than expected. It is likely that simple Center for Innovation protective mechanisms, like mouth guards, could in Pediatric Practice protect against many of these injuries. explored recent suicide This is the first major research publication trends in adolescents resulting from the use of RIO. As the program in the United States is refined and expanded, it will become an using a national increasingly valuable tool for understanding the database. His findings epidemiology and mechanics of injury to high were published in a school athletes and, importantly, how they can peer-reviewed letter to the Journal of the American be prevented. Medical Association, a leading biomedical journal, and were featured in many major national media – Epidemiological features of high school baseball outlets. Significant excess mortality due to youth injuries in the United States, 2005-2007. Pediatrics. 2008 Jun;121(6):1181-7. suicide was found during 2004-2005, suggesting a sustained trend that began the year earlier was Dr. Kim McBride and D not a single-year anomaly. This important work his colleagues in the was a clarion call for additional investigative Center for Molecular work to understand the origin of this disturbing and Human Genetics trend in suicide in the United States. Dr. Bridge have dedicated their is an epidemiologist interested in improving mental careers to understanding health services for children and adolescents. the genetic underpin- – Suicide trends among youths aged 10 to 19 years nings of congenital in the United States, 1996-2005. JAMA. 2008 Sep heart defects, one of 3;300(9):1025-6. the most common birth defects affecting children. The investiga- tive team studied the genetics of left ventricular P A G E 4 :: The Research Institute at Nationwide Children’s Hospital
  • In 2008, faculty in the Division of Neurology contribu made major contribu- tions to the field of pediatric stroke, an important cause of chronic morbidity in children. Dr. Steve Roach was the lead author on a highly visible American Heart Association Scientific Statement on management of stroke in infants and children. This comprehensive document was the first to provide guidelines for diagnosis and treatment of pediatric stroke. Led by Dr. Warren Lo, L a multidisciplinary team including faculty from the Division of Neurology, the N Division of Hematology, the Center for Innovation in Pediatric Practice, and the Center for Biobehavioral Health published the first-ever study of the costs of stroke in children. Complementing an outstanding clinical program in stroke care at Nationwide Children’s Hospital, these scholarly contributions will contribute to improvements in knowledge relating to this under-appreciated morbidity in children. – The cost of pediatric stroke care and rehabilitation. Stroke. 2008 Jan;39(1):161-5. Epub 2007 Nov 21. :: AS A LEADING PEDIATRIC – Management of stroke in infants and children: a scientific statement from a Special Writing Group of BIOMEDICAL RESEARCH the American Heart Association Stroke Council and the Council on Cardiovascular Disease in the Young. INSTITUTION, NATIONWIDE Stroke. 2008 Sep;39(9):2644-91. Epub 2008 Jul 17. Dozens of additional compelling accomplish- CHILDREN’S HOSPITAL IS A ments can be found on The Research Institute’s web site, NationwideChildrens.org/Research. MAJOR CONTRIBUTOR TO NEW KNOWLEDGE THAT WILL LEAD TO IMPROVEMENTS IN THE LIVES OF BOTH ILL AND WELL CHILDREN. The Research Institute at Nationwide Children’s Hospital :: P A G E 5