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Research

Lubiprostone Found to be Safe, as Effective as Placebo in Children With Constipation
 Lubiprostone Found to be Safe, as Effective as Placebo in Children With Constipation 1024 680 Mary Bates, PhD
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Close up color photo of little boy holding hands on his belly

The medication, FDA-approved for adults, may benefit older pediatric patients with less of a behavioral component to their constipation. Pediatric functional constipation, though common, remains challenging to treat. Lubiprostone is a medication approved by the U.S. Food and Drug Administration (FDA) for the treatment of constipation and irritable bowel syndrome with constipation in adults. In…

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Kidney and Urinary Tract Protein Demonstrates Antimicrobial Activity
 Kidney and Urinary Tract Protein Demonstrates Antimicrobial Activity 1024 683 Lauren Dembeck
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A little-studied peptide found in the urinary tract is a potential biomarker for urinary tract infection risk and could be developed into a novel therapeutic against drug-resistant bacteria.  Researchers at Nationwide Children’s Hospital have demonstrated that an understudied protein expressed in the human kidney and bladder kills the bacteria that cause urinary tract infections (UTI).…

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A Novel FUS/ETV4 Fusion and Other Rare FET/ETS Fusion Proteins Are Bona Fide Ewing Sarcoma
 A Novel FUS/ETV4 Fusion and Other Rare FET/ETS Fusion Proteins Are Bona Fide Ewing Sarcoma 1024 575 Jessica Nye, PhD
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Researchers conduct a molecular comparison between the established EWS/FLI translocation with rarer FET/ETS fusions, broadly supporting the inclusion of FET/ETS chimeric proteins in Ewing Sarcoma diagnosis and treatment.   Ewing sarcoma, most commonly caused by a chromosomal translocation of the amino-terminal domain of EWSR1 with the transcription factor FLI (EWS/FLI), presents as an aggressive pediatric…

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Identifying Early Urinary Biomarkers of Kidney Damage in Type 1 Diabetes
 Identifying Early Urinary Biomarkers of Kidney Damage in Type 1 Diabetes 1024 575 Mary Bates, PhD
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Markers of renal tubule injury and oxidative stress indicate diabetic nephropathy before microalbuminuria. Diabetic nephropathy is a common complication of type 1 diabetes. Traditionally, measurement of microalbuminuria is used to screen children with diabetes for diabetic nephropathy. However, evidence suggests that damage to the kidneys may begin soon after diagnosis, much earlier than microalbuminuria can…

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Understanding Black Youth Suicide: Steps Toward Prevention
 Understanding Black Youth Suicide: Steps Toward Prevention 1024 596 Abbie Roth
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To understand the how and why of rising rates of Black youth suicide, researchers call for a ‘Ground Zero’ approach. In December 2019, the Congressional Black Caucus released the statement “Ring the Alarm: The Crisis of Black Youth Suicide in America.” The statement focused on rising suicide rates among Black youth in the United States.…

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Indicators of Blood Clot Potential Directly Relate to Nephrotic Syndrome Severity
 Indicators of Blood Clot Potential Directly Relate to Nephrotic Syndrome Severity 150 150 Katie Brind'Amour, PhD, MS, CHES
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Researchers in the NEPTUNE network and collaborators at Nationwide Children’s Hospital have confirmed their preclinical studies demonstrating a direct correlation between the severity of disease and prothrombotic biomarkers in patients with nephrotic syndrome. Nephrotic syndrome (NS) dramatically increases the risk of a life-threatening blood clot, but until recently, clinicians have had no reasonable biomarker to…

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Targeting DIPG: The Most Puzzling of Pediatric Brain Tumors
 Targeting DIPG: The Most Puzzling of Pediatric Brain Tumors 1024 629 Katie Brind'Amour, PhD, MS, CHES
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Survival has dramatically improved for numerous pediatric cancers over the last several decades, with a notable and very deadly exception: diffuse intrinsic pontine glioma (DIPG). Now, a community of researchers and clinician-scientists have set the stage for a renewed — and better-armed — assault against this beast of a brain tumor. The past 50 years…

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Lessons Learned Through a Global Pandemic
 Lessons Learned Through a Global Pandemic 1024 683 Lauren Bakaletz, PhD
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Lauren Bakaletz, PhD

As a microbiologist and vaccinologist, I spend nearly every day thinking about viruses and bacteria and the diseases they cause, as well as how to best prevent them from doing so. While in graduate school, we were taught about the great ‘flu’ pandemic of 1918 that infected one-third of the world’s population and killed 20-50…

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Lauren Bakaletz Named 2021 Allen Distinguished Scholar in Pediatric Research
 Lauren Bakaletz Named 2021 Allen Distinguished Scholar in Pediatric Research 1024 575 Abbie Roth
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Lauren Bakaletz, PhD, director of the Center for Microbial Pathogenesis in the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital, was named the 2021 Allen Distinguished Scholar in Pediatric Research. The award is given in honor of former Nationwide Children’s CEO, Steve Allen, MD, and his role in growing the AWRI into a preeminent research institution.…

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Boosting Natural Killer Cells for the Treatment of COVID-19
 Boosting Natural Killer Cells for the Treatment of COVID-19 150 150 Lauren Dembeck
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Natural killer cells — critical for immune surveillance and host defense — have an important role in cancer immunotherapy and antiviral responses. While driving to the laboratory one day, Brian Tullius, MA, MD, a fourth-year bone marrow transplant and cell therapies fellow at Nationwide Children’s in the Lee Lab, had an epiphany. “I thought, ‘wait…

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Cerebral Organoids Provide Insight into Human Brain Development and Neurological Disease
 Cerebral Organoids Provide Insight into Human Brain Development and Neurological Disease 1024 575 JoAnna Pendergrass, DVM
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microscopic image of brain organoid

Correlations between the neuroelectrical maturation and cellular development of cerebral organoids highlight the organoids’ translational and therapeutical potential in early neurodevelopmental disorders. Cerebral organoids are three-dimensional miniature organs that resemble the human brain. Derived from human pluripotent stem cells, cerebral organoids have emerged to advance stem cell research, improve three-dimensional tissue culture techniques and enhance…

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Advances in Neonatal GERD
 Advances in Neonatal GERD 1024 683 Mary Bates, PhD
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Dr. Jadcherla

New studies from the Jadcherla Lab provide insights into diagnosing, classifying and treating GERD in infants. Differentiating gastroesophageal reflux (GER), which is defined as the passage of gastric contents into the esophagus, from GER disease (GERD), when reflux is associated with troubling symptoms, remains a challenge in infants. Symptom-based diagnosis and treatment of GERD has…

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Race Is a Risk Factor for Postoperative Death in Apparently Healthy Children in United States
 Race Is a Risk Factor for Postoperative Death in Apparently Healthy Children in United States 1024 683 Abbie Roth
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African American children were nearly 3.5 times more likely to die within 30 days after surgery, compared to white peers. In a new study, published in Pediatrics, researchers have shown that being African American was strongly associated with a higher risk of postoperative complications and mortality among apparently healthy children. In fact, compared to their white peers,…

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Clinical Trials for Blood Cancers: Advancing Options for Children and Young Adults
 Clinical Trials for Blood Cancers: Advancing Options for Children and Young Adults 1024 683 Abbie Roth
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Most cancer treatments are first discovered, developed and approved for adult patients. But translating those therapies to pediatric and young adult patients with cancers can be more of a winding path than a superhighway. At Nationwide Children’s Hospital, the Division of Hematology, Oncology and Blood and Marrow Transplant is dedicated to participating in and supporting clinical…

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Hemophilia Gene Therapy Trials Aim to Reduce Patient Burden
 Hemophilia Gene Therapy Trials Aim to Reduce Patient Burden 1024 495 Katie Brind'Amour, PhD, MS, CHES
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blood cells

Clinical trials using gene therapy to boost the body’s production of clotting factor aim to remove the need for regular infusions — ideally giving patients years or even decades free from daily worry about their condition. Unlike most pediatric treatment centers, Nationwide Children’s Hospital is one of only a few dozen sites worldwide selected to…

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Study Finds Rising Rate of Mental Health Visits Among Youth to Emergency Departments
 Study Finds Rising Rate of Mental Health Visits Among Youth to Emergency Departments 480 320 Kaitlin Hall
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Highest jump was among 15- to 17-year-olds and more pronounced in girls; Dramatic increases in self-harm and substance use as reasons for ED visits. While the number of pediatric emergency department (ED) visits across the nation has remained stable over the last 10 years, visits for mental health disorders have risen 60% and the rate…

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Transforming Medical Science Through Research Affinity Groups
 Transforming Medical Science Through Research Affinity Groups 150 150 Katie Brind'Amour, PhD, MS, CHES
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Purposeful synergy drives the most meaningful medical science. Nephrology physician-scientists require tissue samples, urologists desire tests to know which patients truly require surgery, and basic scientists must find ways to meaningfully assess new animal models to yield clinically valuable data. If each of these professionals had a defined network of colleagues from the other disciplines…

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Epigenetics, Chromatin Architecture and a Judo Mechanism to Attack Cancer
 Epigenetics, Chromatin Architecture and a Judo Mechanism to Attack Cancer 1024 575 Abbie Roth
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Researchers broaden the understanding chromatin architecture in human disease. Epigenetics is the study of how genetic information is context-dependent: it is organized so it can be repressed, but also read, repaired and replicated. For example, transcription factors can “communicate” with each other through the chromatin-DNA interface, and work in combinations to regulate which genes are…

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Unlocking the Structure of Biofilms
 Unlocking the Structure of Biofilms 1024 575 Kevin Mayhood
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Illustration showing the precise pattern of a biofilm - a perfect, 3D matrix

Researchers characterize a component that stabilizes biofilms, a step toward learning ways to disrupt protection of harmful bacteria. In the extracellular DNA lattice of bacterial biofilms, nature appears to reprise the functional equivalent of Holliday junction (HJ) intermediates — cross-shaped structures formed during the process of genetic recombination, researchers at Nationwide Children’s Hospital report in Proceedings…

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Better Bone Healing by Reversing Current Techniques?
 Better Bone Healing by Reversing Current Techniques? 1024 575 Katie Brind'Amour, PhD, MS, CHES
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Bones may heal denser and stronger when given room for controlled micro-movement at first, followed by rigid stabilization — a complete flip-flop of the standard of care. A combination of biology and mechanical influence determines how well a bone heals, for better or worse. For half a century, physicians have believed that complete bone immobilization…

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From Lab Work to “Home Work:” Tips on the Transition to Work From Home
 From Lab Work to “Home Work:” Tips on the Transition to Work From Home 1024 683 Adrianna Matos-Nieves
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PhD candidate Adrianna Matos-Nieves shares tips for research employees who are suddenly finding themselves transitioning from the wet lab to their home office. PhD students have a significant advantage when enduring coronavirus-imposed social distancing. We decided to do it voluntarily many years prior. But in all seriousness, the transition to working from home can be…

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Study Shows Promising Host-Targeted Approach for the Prevention and Cure of Gonorrhoea in Women
 Study Shows Promising Host-Targeted Approach for the Prevention and Cure of Gonorrhoea in Women 1024 683 Nationwide Children's
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In research published in mBio, researchers from the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital and Griffith University’s Institute for Glycomics have discovered non-antibiotic (host-targeted) therapies for the effective treatment of Neisseria gonorrhoeae infections by repurposing existing drugs. Gonorrhoea is the second most commonly reported sexually transmitted infection (STI). The Centers for Disease Control and…

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How Does a Children’s Hospital Excel in the Discovery and Development of New Therapies?
 How Does a Children’s Hospital Excel in the Discovery and Development of New Therapies? 1024 575 Abbie Roth
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conceptual art of DNA

A conversation with Dennis Durbin, MD, MSCE chief scientific officer, Abigail Wexner Research Institute, Nationwide Children’s Hospital Nationwide Children’s has become an epicenter for gene therapy discovery and development. The discovery in 2009 that adeno-associated virus (AAV) could cross the blood-brain barrier was a milestone in the development of dozens of gene therapy products for neuromuscular…

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From Natural Killer Cells to Virus-Specific T Cells: What’s Next in Cellular Therapy
 From Natural Killer Cells to Virus-Specific T Cells: What’s Next in Cellular Therapy 969 533 Abbie Roth
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Illustration of NK Cells, T Cells, other immune cells floating across white background

Harnessing the power of the immune system to overcome cancer, improve outcomes of bone marrow transplants and fight viral infections in immunocompromised pediatric patients is at the heart of cell therapy research.   Natural Killers in Action Natural killer (NK) cells are a component of the innate immune system that mediates the death of cancer…

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Redirecting the Natural Immune Response to Disrupt Bacterial Biofilms
 Redirecting the Natural Immune Response to Disrupt Bacterial Biofilms 150 150 Lauren Dembeck
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A new vaccine candidate has the potential to improve outcomes for patients with chronic, recurrent diseases, such as ear infections. Could this platform technology be an important key in solving the antibiotic resistance threat? Most bacterial species prefer to live in biofilms, where they are protected from antibiotic treatments and can lead to chronic and recurrent…

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The Search to Identify Tumor Cells Evading Chemotherapy
 The Search to Identify Tumor Cells Evading Chemotherapy 150 150 Sanjana Rajan
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Graduate research associate Sanjana Rajan shares why her work to label and track cells before and after chemotherapy is the next step to preventing tumor relapse. For a long time, the cells within a tumor were thought to be similar to one another, like a bowl of chocolate chips. However, scientific studies have identified that…

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RNase 7: Paving the Way for a Natural, Antibiotic-Free Treatment for Urinary Tract Infections
 RNase 7: Paving the Way for a Natural, Antibiotic-Free Treatment for Urinary Tract Infections 150 150 Katie Brind'Amour, PhD, MS, CHES
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The latest in the body of antimicrobial peptide research suggests RNase7 may be a useful prognostic marker and potential therapeutic option for UTIs. Building on their body of research focused on the naturally occurring antimicrobial peptides in the urinary tract, clinician-scientists at Nationwide Children’s Hospital have now confirmed the suspected role of Ribonuclease 7 (RNase…

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How Research Reinforces the Collaborative Culture of a Children’s Hospital
 How Research Reinforces the Collaborative Culture of a Children’s Hospital 1024 575 Steve Allen, MD
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Steve Allen, MD

As he retires, the CEO of Nationwide Children’s Hospital reflects on the less-obvious effects of scientific discovery. Nationwide Children’s Hospital has undergone a dramatic transformation in the last two decades, from an important regional resource into a nationally preeminent medical system. One of the clearest signs of our growth, and one that we’re particularly proud…

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Bacteria Hiding Out Inside Epithelial Cells May Promote Recurring Ear Infections
 Bacteria Hiding Out Inside Epithelial Cells May Promote Recurring Ear Infections 1024 575 Rachael Hardison
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Middle ear infections, also known as otitis media (OM), remain a health care concern for children in the United States and across the world, despite recent therapeutic and technological advances. For the subset of children with chronic or recurring infections, OM becomes a significant socioeconomic burden for caregivers. Chronic or recurrent episodes of OM can…

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Researchers Identify Proteins Triggering Imbalance of Cells in Chronic Lung Disease
 Researchers Identify Proteins Triggering Imbalance of Cells in Chronic Lung Disease 1024 575 Kevin Mayhood
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Illustration of lungs on blue silhouette of upper chest on black background

A protein that triggers an imbalance of mucous and ciliated cells in patients with chronic lung disease could be a target for treatments to restore airways. Many chronic lung diseases in children and adults have one thing in common: the airway lining that normally traps and sweeps out bacteria, viruses and diesel particulates stops functioning…

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Can We Prevent Future Language Delays in the NICU?
 Can We Prevent Future Language Delays in the NICU? 150 150 Abbie Roth
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Researchers investigate the use of event related potentials to measure the effects of mother’s voice exposure on speech sound differentiation. Preterm infants are at high risk for neurosensory impairments and developmental delays, including hearing loss, which may have lasting consequences. Compared to babies born at term, preterm infants are twice as likely to have a…

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Mechanism for Expulsion of DNA from NTHI Described
 Mechanism for Expulsion of DNA from NTHI Described 150 150 Abbie Roth
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Researchers at Nationwide Children’s publish breakthrough discovery revealing how DNA and DNABII proteins are released into the biofilm matrix. Nontypeable Haemophilus influenzae (NTHI) bacteria, a common culprit in otitis media, are known for their ability to create dense biofilms. As the subject of much biofilm and vaccine research, they are increasingly understood as complex and surprising organisms.…

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Meaningful Improvement of Child Health Requires Core Quality and Outcomes Measures
 Meaningful Improvement of Child Health Requires Core Quality and Outcomes Measures 150 150 Brianne Moore
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The seemingly infinite indicators large pediatric systems use to measure children’s health often fall short in determining patient outcomes and quality of care. In an editorial published in JAMA Pediatrics, Kelly Kelleher, MD, director of the Center for Innovation in Pediatric Practice at Nationwide Children’s Hospital, and co-author William Gardner from the Children’s Hospital of Eastern Ontario, call for…

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Different Patterns of Executive Function Deficits in Different Pediatric Brain Disorders
 Different Patterns of Executive Function Deficits in Different Pediatric Brain Disorders 150 150 Mary Bates, PhD
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Clinical implications include specialized testing for deficits in executive function as these may not be apparent at a routine exam. A large group of researchers, including many from Nationwide Children’s Hospital, recently looked at whether children with different brain disorders show diverse patterns of strengths and weaknesses in executive functions. Children with traumatic brain injuries…

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Immune Profiling Leads to Implications for Immunotherapy for NF1-Associated Tumors
 Immune Profiling Leads to Implications for Immunotherapy for NF1-Associated Tumors 150 150 Abbie Roth
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Profiling reveals histologic subtype distinctions and heterogeneity of neurofibromatosis type 1-associated tumors. Neurofibromatosis type 1 (NF1) — an autosomal dominant disorder affecting approximately one in every 3,500 people — results in dysfunctional neurofibromin, a protein expressed throughout the body and involved in the RAS signaling pathway. Virtually all patients with NF1 develop benign peripheral nerve…

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Combining Immunotherapies Effective Against Mouse Model of Cancer
 Combining Immunotherapies Effective Against Mouse Model of Cancer 150 150 Mary Bates, PhD
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Recent study results suggest that combining virotherapy and PD-1 blockades may be more effective than either approach alone. Rhabdomyosarcoma, a cancer made up of cells that normally develop into skeletal muscles, is the most common soft tissue cancer in children. If it is detected early and localized in certain areas, rhabdomyosarcoma is usually curable with…

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Needle-free Immunization Prevents Experimental Otitis Media
 Needle-free Immunization Prevents Experimental Otitis Media 1024 575 Tiasha Letostak, PhD
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Close up, color image of someone extending a gloved hand with a small patch face up on their pointer finger that resembles a circular adhesive bandage that has a smaller, blue square patch on its center

The first data to demonstrate the efficacy of a simple, needle-free vaccine delivery system for middle ear infections. Otitis media (OM), or middle ear infection, accounts for approximately 30 million doctor visits a year in the U.S. The pathogen nontypeable Haemophilus influenzae (NTHI) causes the majority of cases of ear infection, including chronic OM, recurrent OM, and…

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Inside the Good Manufacturing Production Facility
 Inside the Good Manufacturing Production Facility 1024 575 Tiasha Letostak, PhD
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By removing a barrier to access – production of pharmaceutical products for clinical trial – the team at the GMP facility is bringing more treatments to rare diseases. A newly designed, 7,500 square foot clean room suite with multi-use viral vector and cell therapy capabilities is expediting the bench-to-bedside process of bringing life-saving treatments to…

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Discovery to Drug Development: Expanding the Role of Academic Centers
 Discovery to Drug Development: Expanding the Role of Academic Centers 1024 575 Abbie Roth
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As more researchers at academic centers become involved in drug development, institutions are responding with support and guidance. Researchers at academic institutions regularly make discoveries about disease processes and potential therapeutic agents. Translational medicine is focused on moving these discoveries out of the laboratory and into the clinic where they can potentially help patients. But…

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Why Patients With Diabetes Have More UTIs
 Why Patients With Diabetes Have More UTIs 150 150 Mary Bates, PhD
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Insulin regulates an antimicrobial peptide that is suppressed in patients with diabetes. A recent study led by clinicians and researchers at Nationwide Children’s Hospital reports a potential explanation for why urinary tract infections (UTIs) are so common in patients with diabetes – insulin regulates an antimicrobial peptide (AMP) that is suppressed in those patients. Patients…

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What Do Space Rocks Have to Do With Preterm Birth?
 What Do Space Rocks Have to Do With Preterm Birth? 150 150 Abbie Roth
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A brief history of nanobacteria and their implications for human health. I remember when nanobacteria were a really big deal. Press-conference-by-POTUS-about-evidence-of-extraterrestrial-life-level big deal. I hadn’t thought much about them until recently, when they made a surprise appearance in a presentation on idiopathic preterm birth by Irina Buhimschi, MD, director of the Center for Perinatal Research at The…

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Stronger Together: A Multi-institutional Database Is Connecting Down Syndrome Clinics for Better Outcomes
 Stronger Together: A Multi-institutional Database Is Connecting Down Syndrome Clinics for Better Outcomes 1024 575 Stephanie Santoro, MD
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Due to medical advances, people with Down syndrome are living longer than ever before. This increased life expectancy has nearly doubled in the past 25 years. The National Down Syndrome Society estimates that 400,000 people with Down syndrome are living in the United States. Despite this increased life expectancy, little current information on the secondary…

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Solving the Problem of Managing Big Genomic Data
 Solving the Problem of Managing Big Genomic Data 150 150 Abbie Roth
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Researchers at Nationwide Children’s Hospital complete a first-of-its-kind project to evaluate a large-scale genomic data management system on the scale of up to one million genomes. The influx of genomics data resulting from the increasing affordability of whole exome/genome sequencing and President Obama’s Precision Medicine Initiative requires a novel technological solution to data storage, communication…

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Best Practices for Research Recruitment and Retention
 Best Practices for Research Recruitment and Retention 1024 575 Tiasha Letostak, PhD
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You can’t obtain study data without participants. From initial design and promotion to communication tactics and patient satisfaction, here are some strategies to ensure success. Advancing pediatric research depends on successful recruitment and retention of study participants. Unfortunately, 9 out of 10 trials end up having to double their original timelines in order to meet…

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Changing the Game: Virtual Reality Distracts From Pain, Transforming the Patient Experience
 Changing the Game: Virtual Reality Distracts From Pain, Transforming the Patient Experience 844 487 Gina Bericchia
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A first-of-its-kind virtual reality experience from the hemophilia team and design experts at Nationwide Children’s Hospital and The Ohio State University distracts patients with an immersive environment of penguins, pirates and hermit crabs during infusions and other procedures. A pilot study is testing the feasibility of integrating the virtual reality technology into the clinic setting.…

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Oligodendrocytes Induce Motor Neuron Death in ALS
 Oligodendrocytes Induce Motor Neuron Death in ALS 150 150 Jeb Phillips
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A first-of-its-kind oligodendrocyte in vitro model shows that human cells normally supportive of motor neuron function play an active role in amyotrophic lateral sclerosis pathogenesis – and this discovery may point the way toward therapeutic timing and targets. A number of studies over the last decade have shown that cells which normally support motor neurons,…

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Specialized Test Detects Bacterial Infections in Youngest Infants with Fever
 Specialized Test Detects Bacterial Infections in Youngest Infants with Fever 150 150 Tiasha Letostak, PhD
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The diagnostic test has potential to prevent painful testing, unnecessary antibiotics and hospitalizations for many of the more than 500,000 febrile infants who arrive at hospitals each year. Physicians from Children’s Hospital of Michigan, UC Davis Medical Center and Nationwide Children’s Hospital, in collaboration with 19 other pediatric emergency departments around the country, have established…

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Using Whole Exome Sequencing to Find Genetic Cause of Congenital Heart Disease in At-Risk Patients
 Using Whole Exome Sequencing to Find Genetic Cause of Congenital Heart Disease in At-Risk Patients 150 150 Brianne Moore
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Whole exome sequencing has the ability to identify disease-causing mutations, contributing to the development of personalized medicine and bridging a crucial gap between scientific knowledge and clinical application. Congenital heart disease (CHD) is the most common type of birth defect, affecting approximately 40,000 births per year in the United States. While some types of CHD…

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Study Finds Genetic Loci Associated With Anxiety and Aggression in Dogs: What Does It Mean for Kids?
 Study Finds Genetic Loci Associated With Anxiety and Aggression in Dogs: What Does It Mean for Kids? 150 150 Carlos E. Alvarez, PhD
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Principal investigator Carlos Alvarez, PhD, reports the genome wide mapping of nine fear and aggression traits in dogs and discusses the implications of the findings. Anxiety disorders are the most common type of mental illness in the United States. They are associated with increased risk of schizophrenia, depression, addiction and other psychiatric disorders. While much…

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