Medical Professional Publications

Research Rap - February 2009

Nationwide Children’s Elected Leader of Cystic Fibrosis Translational Research

A new designation will allow Nationwide Children’s Hospital to lead cutting-edge research combating the lifeshortening, genetic disease cystic fibrosis. Among 116 specialized cystic fibrosis centers throughout the country, Nationwide Children’s has been selected to serve as one of 13 translational research centers for the Cystic Fibrosis Foundation’s Therapeutics Development Network (CFF TDN). These translational research centers will lead the newest Phase I clinical trials and provide scientific direction to the entire network.

Historically, the CFF TDN has been comprised of 18 clinical research centers intended to fast-track improvements in cystic fibrosis care by streamlining the research in an environment of patient safety and high standards of research consistency. In order to meet the growing need for study participants and expanded research programs, a decision was made in 2007 to expand the network to include 64 “routine research centers” and 13 translational research centers.

The Section of Pulmonary Medicine at Nationwide Children’s participated in its first cystic fibrosis clinical trial in 1992, the third-ever cystic fibrosis clinical trial world-wide, and has since taken part in more than 50 clinical trials for cystic fibrosis. The hospital has been a CFF TDN member since 2002.

Non-Invasive Method Delivers Genes to the Central Nervous System

Investigators at The Research Institute have identified a non-invasive method for delivering genes to the central nervous system, a strategy that penetrates the body’s protective blood-brain barrier with unprecedented success.

The blood-brain barrier (BBB) is a protective network of blood vessels and cells that prevents many substances from entering brain tissue and the central nervous system. While the BBB functions as a vital biological shield, it can also serve as a barrier to treating central nervous system disorders.

Previous gene therapy efforts to treat two of the most common motor neuron diseases, spinal muscular atrophy (SMA) and ALS (Lou Gehrig’s disease), have either failed to bypass the BBB, or are clinically irrelevant since they require interventions considered too numerous or too complicated.

In this study*, which appears in Nature Biotechnology, members of the Center for Gene Therapy at The Research Institute used AAV9, a subtype of the adeno-associated virus, as the vehicle for gene transfer. Additionally, new techniques stemming from this AAV9 research could target neurons in adults. Such strategies could provide new treatment options for diseases such as Huntington’s that involve multiple brain regions.

*Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol. 2008 Dec 21. [Epub ahead of print]

Shoulder Injuries in U.S. High School Athletes Occur More Often in Boys

Although shoulder injuries accounted for just eight percent of all injuries sustained by high school athletes, shoulder injuries were relatively common in predominately male sports such as baseball (18 percent of all injuries), wrestling (18 percent) and football (12 percent). Moreover, boys experienced higher shoulder injury rates
than girls, particularly in soccer and baseball/softball.

Player-to-player contact was associated with nearly 60 percent of high school athletes’ shoulder injuries from 2005 through 2007, according to a study published in the online issue of the Journal of Athletic Training and conducted by researchers in the Center for Injury Research and Policy of The Research Institute at Nationwide Children’s Hospital. This is the first study to examine shoulder injuries across sports in a nationally representative sample of U.S. high school athletes.

The most common shoulder injuries included sprains and strains (37 percent), dislocations and separations (24 percent), contusions (12 percent) and fractures (7 percent). Surgery was required for 6 percent of shoulder injuries. Dislocations and separations accounted for more than half of all shoulder surgeries. Learn more about the Center for Injury Research and Policy.

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