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Exploring the Complex Immune Microenvironment of Rasmussen Encephalitis
 Exploring the Complex Immune Microenvironment of Rasmussen Encephalitis 1024 587 Lauren Dembeck
By: Lauren Dembeck

Researchers used a multi-omics approach to gain a deeper understanding of aberrant cellular signaling leading to progression of a rare childhood neurological disease.   Rasmussen encephalitis is a very rare childhood neurological disease characterized by chronic progressive inflammation of one hemisphere of the brain, leading to loss of function, neurodegeneration, and drug-resistant epilepsy. Researchers have…

Low-Resource Screening Tool Demonstrates Superior Predictive Value for Cerebral Palsy in Infants
 Low-Resource Screening Tool Demonstrates Superior Predictive Value for Cerebral Palsy in Infants 1024 683 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

The use of lower-resource screening tools for cerebral palsy in infants can help increase early detection of the condition and promote access to early interventions.   A recent study reported that the Hammersmith Infant Neurological Examination (HINE), when compared with the Test of Infant Motor Performance (TIMP), had the better predictive value for detecting cerebral…

Real-World Urinary Management and Outcomes in Cloacal Exstrophy Among Children and Adults
 Real-World Urinary Management and Outcomes in Cloacal Exstrophy Among Children and Adults 1024 683 Jessica Nye, PhD
By: Jessica Nye, PhD
Molly Fuchs, MD

Most (94%) older children and adults with cloacal exstrophy are managed by incontinent diversion or clean intermittent catheterization. “The published literature about cloacal exstrophy can be misleading, and there is an impression that with surgical bladder reconstruction, normal bladder function and voiding can be restored. That is to say, a child will be able to…

Supplementation With Omega 3-6 Improves a Marker of Inflammation Among Children Newly Diagnosed With Autism Spectrum Disorder
 Supplementation With Omega 3-6 Improves a Marker of Inflammation Among Children Newly Diagnosed With Autism Spectrum Disorder 1024 681 Jessica Nye, PhD
By: Jessica Nye, PhD

Fatty acid supplementation may be a treatment route for addressing some underlying causes of autism spectrum disorder (ASD) among children.   One potential mechanism affecting autism-related behaviors is inflammation. In general, ASD severity has been associated with inflammatory cytokine levels and externalizing behaviors with interleukin (IL)-10 and IL-1β levels. Supplementation may be a treatment route,…

CFTR Modulation Therapy Improves Markers of Inflammation and Lung Function in Cystic Fibrosis
 CFTR Modulation Therapy Improves Markers of Inflammation and Lung Function in Cystic Fibrosis 1024 575 Jessica Nye, PhD
By: Jessica Nye, PhD
Illustration of lungs on blue silhouette of upper chest on black background

Six months of elexacaftor-tezacaftor-ivacaftor (ETI) therapy significantly reduced proinflammatory cytokines, increased immune cell composition, and improved clinical outcomes in cystic fibrosis (CF).   “Cystic fibrosis is a progressive lung disease that carries significant morbidity,” says Shahid Sheikh, MD, of the Pulmonary Medicine and Allergy/Immunology section at Nationwide Children’s Hospital and a professor at The Ohio…

Predicting Risk of Nosocomial Infection in Pediatric Burn Patients: Can Measures of Adaptive Immune Function Help?
 Predicting Risk of Nosocomial Infection in Pediatric Burn Patients: Can Measures of Adaptive Immune Function Help? 150 150 Abbie Miller
By: Abbie Miller

Pediatric burn injuries affect 1 million children each year in the United States. While burn care improvements have decreased mortality, infection-related complications remain a significant problem. According to the National Burn Repository, four of the top five complications of pediatric burns are infection related. Plus, infection-related complications occur in up to 60% of patients with…

Intrauterine Devices are a First-Line Option for Adolescents with Bleeding Disorders
 Intrauterine Devices are a First-Line Option for Adolescents with Bleeding Disorders 150 150 Mary Bates, PhD
By: Mary Bates, PhD

Among adolescents with heavy menstrual bleeding, study shows no difference in IUD expulsion rate between adolescents with bleeding disorders and those without. In a new study, researchers from Nationwide Children’s evaluated the use of intrauterine devices (IUDs) in two young women’s hematology clinics. They found that young women with bleeding disorders did not experience a…

Biofilms In Respiratory Infections and Beyond: A Review
 Biofilms In Respiratory Infections and Beyond: A Review 150 150 Emily Siebenmorgen
By: Emily Siebenmorgen

Researchers from Nationwide Children’s recently published a landmark piece in Frontiers in Cellular and Infection Microbiology reviewing everything known about biofilm aggregates and their role in respiratory tract infections to date. Biofilms create sticky situations for several diseases. These microbial communities are formed when bacterial cells stick to each other and attach themselves to a…

Featured Researcher — Motao (Matt) Zhu, MS, MD, PhD
 Featured Researcher — Motao (Matt) Zhu, MS, MD, PhD 150 150 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Motao (Matt) Zhu, MS, MD, PhD, serves as a principal investigator in the Center for Injury Research and Policy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital. After starting off his epidemiology studies in reproductive health, Dr. Zhu transitioned into injury research and has since become an internationally recognized expert in young driver…

The Impact and Implications of Interferons Against RSV
 The Impact and Implications of Interferons Against RSV 1024 575 Emily Siebenmorgen
By: Emily Siebenmorgen
Illustration of lungs on blue silhouette of upper chest on black background

Specific cytokines play a key role in minimizing disease severity Respiratory syncytial virus (RSV) shares many symptoms with other respiratory viruses like the common cold or the flu, but it represents the number one cause of hospitalization in infants worldwide. Despite being a large burden on the health care system, there are currently few preventative…

Using Hormone Therapy First Drives Earlier Remission From Infantile Epileptic Spasms Syndrome
 Using Hormone Therapy First Drives Earlier Remission From Infantile Epileptic Spasms Syndrome 1024 575 Natalie Wilson
By: Natalie Wilson

Recent research conducted at Nationwide Children’s Hospital demonstrated standardizing hormone therapy as the first treatment for infantile spasms improved rates of early remission. Infantile epileptic spasms syndrome (IESS) is an uncommon epilepsy syndrome characterized by seizures called infantile spasms that begin between 1 and 24 months of age (with a peak age of onset at…

New Nomogram Helps Optimize 3-D Rotational Angiography for Congenital Cardiac Catheterization
 New Nomogram Helps Optimize 3-D Rotational Angiography for Congenital Cardiac Catheterization 1024 775 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Clinician-researchers studied a large number of 3-D rotational angiography (3DRA) cases to identify patient and practice factors that produce the highest quality images. In an effort to aid in the successful application of 3-D rotational angiography (3DRA) in cardiac catheterization labs worldwide, a team of clinicians at Nationwide Children’s Hospital retrospectively reviewed 208 3DRAs to…

Bowel Management Program Improves Urinary Symptoms Associated with Pediatric Functional Constipation
 Bowel Management Program Improves Urinary Symptoms Associated with Pediatric Functional Constipation 150 150 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

A one-week bowel management program can significantly improve urinary symptoms in children with functional constipation, possibly eliminating the need for extensive urologic testing. Molly Fuchs, MD, a urologist at Nationwide Children’s Hospital, and her research team recently demonstrated that a bowel management program (BMP) significantly improved urinary symptoms in children with functional constipation. Functional constipation…

Development of Acute Kidney Injury as a Potential Biomarker of Necrotizing Enterocolitis in Preterm Infants
 Development of Acute Kidney Injury as a Potential Biomarker of Necrotizing Enterocolitis in Preterm Infants 1024 683 Mary Bates, PhD
By: Mary Bates, PhD

Regular measurement of serum creatinine levels could help identify necrotizing enterocolitis sooner. Researchers at Nationwide Children’s neonatal intensive care unit (NICU) evaluated serum creatinine levels and the development of acute kidney injury as a potential biomarker of necrotizing enterocolitis in preterm infants. The retrospective chart review showed that acute kidney injury is more prevalent in…

Study Suggests Survivors of Pediatric Brain Tumors Have Altered Connectivity of Key Brain Regions
 Study Suggests Survivors of Pediatric Brain Tumors Have Altered Connectivity of Key Brain Regions 1024 575 Lauren Dembeck
By: Lauren Dembeck

The findings contribute to a scant body of literature on the changes in functional connection within the brain and neurocognitive outcomes in survivors of pediatric brain tumors. Pediatric brain tumors are the most common solid tumor diagnosis among children in the United States. With advances in treatment, the 5-year survival rate among these children and…

How Can We Ensure Use of Artificial Intelligence in Pediatric Medicine Is Ethical?
 How Can We Ensure Use of Artificial Intelligence in Pediatric Medicine Is Ethical? 915 341 Jessica Nye, PhD
By: Jessica Nye, PhD

Team diversity, data contextual factors, and transparency are key factors for the ethical use of artificial intelligence (AI) in pediatric medicine. AI has been increasingly used in medicine to help clinicians predict patient risk and make treatment decisions. A discussion about the ethical translation of AI into pediatric medicine was published as a comment in…

Featured Researcher — Brenda Lilly, PhD
 Featured Researcher — Brenda Lilly, PhD 150 150 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Brenda Lilly, PhD, a principal investigator in the Center for Cardiovascular Research at Nationwide Children’s Hospital, examines the interactions between endothelial cells, which line the inside of blood vessels, and smooth muscle cells. Blood vessels play a critical role in a wide variety of diseases. As a basic biologist, Dr. Lilly studies the mechanisms involved…

Do Warning Labels on High-Powered Magnets Prevent Child Injury?
 Do Warning Labels on High-Powered Magnets Prevent Child Injury? 1024 521 Laura Dattner
By: Laura Dattner
cube made of ball magnets in the palm of a hand

A recent study suggests parents often don’t notice or read warning labels on high powered magnets. They also perceive these products as children’s toys, despite FDA rules against marketing them to children under age 14 years. High-powered magnet products are made of rare-earth metals, usually neodymium, that is up to 30 times more powerful than…

Examining Pediatric Injury-Related Emergency Department Vistis During the COVID-19 Pandemic
 Examining Pediatric Injury-Related Emergency Department Vistis During the COVID-19 Pandemic 480 320 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

During the first year of the COVID-19 pandemic, the frequency of pediatric injury-related visits to the emergency department decreased, while injury severity increased. The COVID-19 pandemic caused disruptions not only on a global scale but also for individuals, particularly children. Pandemic-related closures of school and extracurricular activities significantly impacted the daily routines of children and…

Significant Weight Gain Observed in Low-Income Youth During the Early COVID-19 Pandemic
 Significant Weight Gain Observed in Low-Income Youth During the Early COVID-19 Pandemic 150 150 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

Low-income youth experienced significant weight gain during the first six months of the COVID-19 pandemic, highlighting the need for simultaneous childhood obesity prevention and treatment.   The COVID-19 pandemic and subsequent mitigation efforts profoundly affected children. Specifically, remote learning and cancellation of extracurricular activities disrupted children’s daily routines and reduced opportunities for physical activity, increasing…

InSight: Total Pancreatectomy With Islet Autotranplantation: The Basics
 InSight: Total Pancreatectomy With Islet Autotranplantation: The Basics 1024 500 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
illustrated cross section of islets from pancreas

Download a PDF version of this image.       This feature was published in the Fall/Winter 2022 print issue. Download the full issue.  Image credit: Mandy Root-Thompson for Nationwide Children’s

TPIAT: A Way Forward for Chronic Pancreatitis
 TPIAT: A Way Forward for Chronic Pancreatitis 1024 500 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
illustrated cross section of islets from pancreas

Nationwide Children’s Hospital has assembled a veritable “dream team” of pancreatitis and pediatric transplant surgery experts to offer what they hope will become the world’s preeminent pediatric center for complex pancreatic care. Imagine your child suffering from a sudden, debilitating episode of abdominal pain. No obvious cause, no cure, just pain so severe it requires…

How One Family In Appalachia Changed the Medical Field’s Understanding of Pancreatitis
 How One Family In Appalachia Changed the Medical Field’s Understanding of Pancreatitis 150 150 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

More than 30 years ago, a teenager from Kentucky named Kevin Slone had his first attack of acute pancreatitis. Doctors at the Cleveland Clinic urged his father, Bobby Slone, to try to document whether other people in the family had similar stomach problems. Bobby Slone took the homework to heart, documenting incidences of similar symptoms…

Understanding Pediatric Critical COVID-19 Disease Around the World
 Understanding Pediatric Critical COVID-19 Disease Around the World 150 150 Natalie Wilson
By: Natalie Wilson

A large, multinational study found more children hospitalized with critical COVID-19 died than was indicated by previous studies.   Children under 18 represent around 22% of the population in the United States. Yet, just 15% of over 75 million U.S. COVID-19 cases reported to the Centers for Disease Control (CDC) and fewer than 0.2% of over…

Multimodal Molecular Profiles Offer Clinically Valuable Information to Oncologists
 Multimodal Molecular Profiles Offer Clinically Valuable Information to Oncologists 150 150 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Expanding the profiling ordered for central nervous system (CNS) tumors may meaningfully impact diagnosis and treatment. Prajwal Rajappa, MD, MS, a physician-scientist and principal investigator for the Steve and Cindy Rasmussen Institute for Genomic Medicine (IGM) at Nationwide Children’s Hospital, has long advocated for a more robust use of precision medicine approaches to improve clinicians’…

Ewing Sarcoma Transcription Factor Mediates Changes in Transcription by Reprogramming Chromatin Structure
 Ewing Sarcoma Transcription Factor Mediates Changes in Transcription by Reprogramming Chromatin Structure 1024 575 Abbie Miller
By: Abbie Miller
yellow squiggly lines representing chromatin strands in the nucleus

By binding to DNA in the nucleus, the Ewing sarcoma-specific transcription factor EWS/FLI changes chromatin organization, leading to altered gene expression and the development of Ewing sarcoma.   A new report, published in Nucleic Acids Research, focuses on how the Ewing sarcoma-specific transcription factor EWS/FLI changes spatial organization of DNA in the nucleus of Ewing…

Breast Surgery in Adolescents: When Should You Consider It?
 Breast Surgery in Adolescents: When Should You Consider It? 150 150 Ibrahim Khansa, MD
By: Ibrahim Khansa, MD

Research shows that addressing macromastia, breast asymmetry and gynecomastia in adolescence can improve physical symptoms as well as quality of life and mental wellbeing. Breast disorders such as macromastia, breast asymmetry and gynecomastia are in adolescents.2 Those conditions can negatively impair quality-of-life, and their surgical treatment has been shown to improve self-esteem, as well as…

Brain Organoids Provide Novel Mechanistic Insight into Microcephaly Associated with AUTS2 Syndrome
 Brain Organoids Provide Novel Mechanistic Insight into Microcephaly Associated with AUTS2 Syndrome 873 552 Lauren Dembeck
By: Lauren Dembeck
Microscopy image of cerebral organoid sections

Researchers have functionally validated a genotype-phenotype relationship between a variant in the AUTS2 gene and neurodevelopmental deficits in AUTS2 syndrome using patient-derived brain organoids. Genetic variants in more than 100 genes, including the gene AUTS2, have been associated with autism spectrum disorders. However, the model systems used to study the mechanisms underlying how these genetic…

Predicting Response to Bronchodilators in Infants with Bronchopulmonary Dysplasia
 Predicting Response to Bronchodilators in Infants with Bronchopulmonary Dysplasia 1024 683 Mary Bates, PhD
By: Mary Bates, PhD

Study emphasizes importance of identifying those patients who will respond to treatment. Certain pulmonary function test parameters in newborns with bronchopulmonary dysplasia are associated with bronchodilator response, according to a new study from researchers at Nationwide Children’s Hospital. The team also found that responders had evidence of greater dysanaptic lung growth than nonresponders. Bronchodilators are…

Study Shows 1 in 4 Girls Born With Anorectal Malformations Have Co-occurring Gynecologic Anomalies
 Study Shows 1 in 4 Girls Born With Anorectal Malformations Have Co-occurring Gynecologic Anomalies 1024 575 Jessica Nye, PhD
By: Jessica Nye, PhD

Gynecological anomalies are common among girls with anorectal malformations (ARM) with or without associated vertebral, anorectal, cardiovascular, trachea-esophageal, renal and limb (VACTERL) anomalies. One in 5,000 live births have ARM and 20%-70% co-occur with other congenital anomalies, including VACTERL. For girls born with ARM, gynecologic congenital anomalies can occur, especially with more complex ARM. To…

Discovering the Genetic Causes of Congenital Heart Defects
 Discovering the Genetic Causes of Congenital Heart Defects 1024 575 Lauren Dembeck
By: Lauren Dembeck

By studying the genomes of children and members of their family, researchers have discovered novel disease genes in families impacted by cardiac defects. Congenital heart disease is a leading cause of death in children under one year of age, and evidence supports a strong genetic component underlying this group of common developmental defects. However, historically,…

Quality of Life in Pediatric Patients With Non-ambulatory Cerebral Palsy and Epilepsy
 Quality of Life in Pediatric Patients With Non-ambulatory Cerebral Palsy and Epilepsy 600 400 Mary Bates, PhD
By: Mary Bates, PhD

Study indicates that quality of life in young people with non-ambulatory cerebral palsy decreases with the use of more antiseizure medications. In a new study, researchers from Nationwide Children’s Hospital assessed the relationship between epilepsy-specific characteristics and proxy-reported health-related quality of life in children and young people with non-ambulatory cerebral palsy. The findings indicate that…

The Latest Developments in Limb Lengthening and Reconstruction Research
 The Latest Developments in Limb Lengthening and Reconstruction Research 1024 683 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
Dr. Iobst with patient

Orthopedic experts publish an invited review of the past year’s most significant papers in the field of limb deformity correction and lengthening. To aid clinicians and surgeons who don’t have time for an annual deep-dive into the literature of their subspecialty, The Journal of Bone & Joint Surgery publishes yearly reviews courtesy of invited guest…

Anal Sphincter Botox Injections for Children With Severe Constipation
 Anal Sphincter Botox Injections for Children With Severe Constipation 1024 680 Mary Bates, PhD
By: Mary Bates, PhD
Close up color photo of little boy holding hands on his belly

The treatment is safe and effective for many children with Hirschsprung disease and, to a lesser extent, functional constipation. In a new prospective study, researchers at Nationwide Children’s evaluated children with severe, refractory constipation treated with anal sphincter botulinum toxin (Botox) injection. They found the treatment can be effective in patients with both Hirschsprung disease…

Long-Term Pulmonary Symptoms Following COVID-19 Infection in Children
 Long-Term Pulmonary Symptoms Following COVID-19 Infection in Children 1024 683 Mary Bates, PhD
By: Mary Bates, PhD

Fatigue and shortness of breath may persist, even in the absence of abnormal test results. Researchers at Nationwide Children’s recently published the largest U.S. study to date of pediatric patients with long-term pulmonary sequelae following COVID-19 infection. The authors note the persistence of symptoms such as shortness of breath and fatigue and highlight at least…

Profiling the Tumor Microenvironment Offers New Insight for Hard-to-Treat Tumors
 Profiling the Tumor Microenvironment Offers New Insight for Hard-to-Treat Tumors 1024 269 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

A multi-faceted approach to precision medicine may allow clinicians to gain a better understanding of cancer and the complex ecosystem in which it resides. The field of precision medicine has evolved in leaps and bounds in the past two decades. Genomic profiling tools that were once cost-prohibitive and slow are now reasonably affordable and fast…

New Disorder of Copper Metabolism Identified, Caused by Variants in CTR1 Gene
 New Disorder of Copper Metabolism Identified, Caused by Variants in CTR1 Gene 150 150 Abbie Miller
By: Abbie Miller

A novel missense mutation in identical twins leads to discovery and definition of new inherited disorder of brain copper metabolism. Copper is essential for many cellular functions, including cellular respiration, antioxidant defense, neurotransmitter biosynthesis and neuropeptide amidation, among others. Until recently, only two inborn errors of copper metabolism were well established. Both are rare. Wilson’s…

How are Depression, Diabetes Distress and Glycemic Control Related in Adolescents With Type 2 Diabetes?
 How are Depression, Diabetes Distress and Glycemic Control Related in Adolescents With Type 2 Diabetes? 150 150 Mary Bates, PhD
By: Mary Bates, PhD

For adolescents with T2D, obesity and attempts at weight loss may contribute more to depression and diabetes distress than the demands of diabetes management. Diabetes distress is a negative emotional response to living with diabetes, a life-threatening illness that requires chronic, demanding, self-management. It is frequently mistaken for depression and the two are interrelated. Previous…

Nephrotoxic Medications in the NICU
 Nephrotoxic Medications in the NICU 1024 575 Mary Bates, PhD
By: Mary Bates, PhD
Nurse caring for infant in Neonatal Intensive Care Unit (NICU)

New study shows treatment with nephrotoxic medications in newborns is associated with the development of acute kidney injury, yet remains common. A multicenter analysis of nearly 50 pediatric hospitals showed that newborns are commonly treated with nephrotoxic medications and, troublingly, the prevalence of acute kidney injury is higher in newborns treated with these drugs. The…

Do Symptom Scores and pH Impedance Correlate in Infants Treated for Gastroesophageal Reflux?
 Do Symptom Scores and pH Impedance Correlate in Infants Treated for Gastroesophageal Reflux? 1024 683 Lauren Dembeck
By: Lauren Dembeck

Gastroesophageal reflux (GER) — the passage of gastric contents into the esophagus — is a physiologic process that can be considered normal or abnormal depending on whether troublesome symptoms and/or complications also occur. In infants, it is difficult for clinicians to distinguish between GER and pathophysiologic GER disease (GERD). “Current diagnostic criteria for GERD are…

Salivary MicroRNAs: A Promising Biomarker for Persistent Post-concussive Symptoms in Children With Concussion
 Salivary MicroRNAs: A Promising Biomarker for Persistent Post-concussive Symptoms in Children With Concussion 1024 768 Lauren Dembeck
By: Lauren Dembeck

The multidisciplinary study used children’s saliva and genomic analysis to identify promising biomarkers for persistent post-concussive symptoms. Predicting clinical recovery of children with concussion is challenging. While concussion symptoms typically resolve within one to three weeks, up to one-third of children develop persistent post-concussive symptoms (PPCS), including headache, nausea, dizziness, blurred vision, and sensitivity to…

Telehealth Has the Potential to Bridge Gaps in Psychiatric and Behavioral Health Care
 Telehealth Has the Potential to Bridge Gaps in Psychiatric and Behavioral Health Care 1024 535 Jessica Nye, PhD
By: Jessica Nye, PhD
Collage of health technology tools

A growing body of evidence supports telebehavioral health delivery for children, adolescents and their families, as outcomes are comparable to in-person treatment delivery. Despite evidence of efficacy, regulatory and financial concerns and technology barriers continue to impede widespread uptake of telebehavioral health. “There have definitely been silver linings to the pandemic, and this is one…

What’s the Scope of Nephritis in Pediatric Patients With IgA Vasculitis?
 What’s the Scope of Nephritis in Pediatric Patients With IgA Vasculitis? 150 150 Jessica Nye, PhD
By: Jessica Nye, PhD

A multicenter study from the Pediatric Nephrology Research Consortium found that many pediatric patients with IgA vasculitis nephritis (IgAVN) are treated with immunosuppressive (IS) agents, and some had evidence of chronic kidney disease as early as 12 months after presentation.   IgA vasculitis (IgAV) is the most common vasculitis in children, usually presenting with typical…

A Single-Dose Cancer Immunotherapy via Gene Transfer
 A Single-Dose Cancer Immunotherapy via Gene Transfer 150 150 Mary Bates, PhD
By: Mary Bates, PhD

Researchers at Nationwide Children’s Hospital report proof-of-principle results for a new gene therapy cancer treatment. The off-the-shelf, single-dose immunotherapy serves as an alternative to CAR-T therapy and can be engineered to be on-demand. T cells redirected to cancer cells either via a chimeric antigen receptor (CAR-T) or a bispecific molecule have been game-changers in treating…

Researchers Characterize Growth and Remodeling of Tissue-Engineered Blood Vessels
 Researchers Characterize Growth and Remodeling of Tissue-Engineered Blood Vessels 600 400 Lauren Dembeck
By: Lauren Dembeck
Tissue engineered vascular graft

Tissue engineering may soon give babies with congenital heart disease new blood vessels capable of native function and growth.   Major cardiovascular reconstructive operations in babies with congenital heart defects require the use of artificial blood vessels called vascular grafts. Because these grafts are made of man-made materials, the recipients’ tissues eventually begin to outgrow…

Predicting Disease Severity and Assessing Long-Term Outcomes of Pediatric La Crosse Virus
 Predicting Disease Severity and Assessing Long-Term Outcomes of Pediatric La Crosse Virus 1024 683 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

Limited data exist on mosquito-borne La Crosse Virus in children, underscoring the importance of determining which patients are at risk for severe disease and long-term neurobehavioral difficulties.   La Crosse Virus (LACV) is a neuroinvasive arbovirus spread by the Aedes triseriatus mosquito, causing symptoms such as vomiting, seizures and altered mental status (AMS). Within the…

Investigators Develop Technique to Effectively Edit NK Cells to Target Specific Cancer Cells
 Investigators Develop Technique to Effectively Edit NK Cells to Target Specific Cancer Cells 1024 649 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
artistic rendering of an NK cell on a faded blue background with other cells faintly visible in the background

The third of three major challenges in enabling the clinical potential of NK cells for cancer therapy has just been addressed, thanks to the combined talents of gene editing experts and NK cell specialists at Nationwide Children’s Hospital.   Originally, NK cells seemed promising for a natural therapy to fight cancer due to their innate…

How Do Children With IBD and Their Caregivers Perceive Biosimilars?
 How Do Children With IBD and Their Caregivers Perceive Biosimilars? 640 427 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

Many pediatric patients with inflammatory bowel disease and their caregivers are unaware of biosimilars, creating an opportunity for providers to educate patients and their families about this effective and less expensive treatment option. Infliximab is a biologic therapy that effectively manages inflammatory bowel disease (IBD) in adult and pediatric patients. However, biologic therapies are expensive,…

Functional Neurological Disorders: What Pediatric Neurologists Should Know
 Functional Neurological Disorders: What Pediatric Neurologists Should Know 150 150 Mary Bates, PhD
By: Mary Bates, PhD

The diagnosis is common, yet this group of disorders has been subjected to confusion and stigma throughout history. Functional neurological disorders are a collection of disorders in which patients experience neurological symptoms, such as numbness, weakness, seizure-like events, or abnormal gait or movements, but without a visible underlying pathology. Underlying mechanisms for these disorders are…

Self-Harm Risk Among Youth With First-Episode Psychosis
 Self-Harm Risk Among Youth With First-Episode Psychosis 1024 575 Mary Bates, PhD
By: Mary Bates, PhD

Study narrows down period of acute risk and finds shared and unique risk factors among adolescents and young adults. Although it is known that young people with psychosis are at an elevated risk for suicide and suicide-related behavior, it has not been clear which patients are at the most risk and when. In a new…