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Research by Division
Clinical and translational research is conducted by each of the divisions in the Department of Pediatrics. Follow the links below to get details about clinical and translational efforts in each:
- Allergy and Immunology
- Cardiology
- Child Abuse and Neglect
- Critical Care Medicine
- Emergency Medicine
- Endocrinology
- General Pediatrics and Adolescent Health
- Hematology-Oncology
- Hospital Medicine
- Infectious Diseases
- Medical Genetics
- Medicine-Pediatrics
- Neonatology
- Nephrology
- Neurology
- Pulmonary and Sleep Medicine
- Rheumatology
- Transplant and Cellular Therapy
Basic Science & Translational Research
Our basic science and translational researchers build upon our highly successful clinical care programs and linkages with other researchers throughout the School of Medicine and Duke University to make fundamental discoveries that will help diagnose, treat and prevent illnesses impacting the health and well being of children. Their laboratories also serve as sites for the training of tomorrow's clinician-scientists and academic leaders who will innovate and discover new ways of caring for children.
The following Department of Pediatrics principal investigators are currently conducting basic science research:
Research Laboratories
Our group is interested in diseases of the liver, ranging from viral hepatitis to metabolic liver disorders and liver cancer. The lab specializes in human liver chimeric mice and created the first xenograft model for metabolic liver disease as well as a novel patient derived cancer model. These humanized mouse models will help to advance and improve experimental therapies for liver disease.
As a neonatologist, my research interests revolve around improving the survival and quality of life of high-risk neonates cared for in Neonatal Intensive Care Units. My primary interest is perinatal brain injuries impacting both full-term infants and those born prematurely. One of the most common forms of perinatal brain injury involves damage to white matter (myelin). My laboratory has developed models of perinatal brain injury to investigate how the endogenous neural stem cell responds to myelin injury.
Newborn infants, especially premature infants, are at risk for severe infections. This susceptibility to infection is a consequence of the unique nature of the newborn's immune system, which is still in the process of learning how to create protective immune responses against disease-causing microbes, while maintaining immune tolerance to self. My research focuses on T cells, and how they function in the developing immune system. Specifically, I am interested in how T cells are able to maintain immune tolerance to oneself, while also protecting against infection. By understanding these cells and their function, we hope to develop targeted therapies that will help newborn babies and older children to prevent and fight infections. As a neonatologist, my greatest hope is that our work will lead to changes in practice that help to improve the survival and quality of life for our smallest patients.
Our research involves identifying gene regulatory elements across the genome to help us understand how chromatin structure dictates cell function and fate. For the last 25 years, mapping DNase I hypersensitive sites has been the gold standard method to identify the location of active regulatory elements, including promoters, enhancers, silencers, and locus control regions. We have developed two technologies that can identify most DNase I hypersensitive sites from potentially any cell type from any species with a sequenced genome. These data can be combined with other wet-lab and computational data types to better understand how these regulatory regions control global gene expression.
Dr. Deel is a clinician scientist in Pediatric Hematology/Oncology. In addition to caring for pediatric patients with hematologic disorders or malignancy, his research focuses on finding novel ways to target fusion-positive pediatric sarcomas. His current work focuses on understanding the gene regulation and molecular pathways responsible for alveolar rhabdomyosarcoma, which is among the most difficult to cure pediatric cancers.
Our laboratory focuses on the control of breathing and pulmonary mechanics in murine models of several genetic diseases. We are currently focuased on ALS mouse models and the Pompe disease mouse model. We examine the impact of gene therapy and neuromodulation on respiratory insufficiency that results from neuromuscular weakness. As a clinician scientist, my goal is to bring therapy from the bench to the bedside and enhance our research at the bench through observations at the bedside.
Dr. Gbadegesin's research is focused on understanding the critical pathways that are involved in the pathogenesis of nephrotic syndrome and in congenital malformations of the kidney and the urinary tract. He and his group have established a phenotypic and biorepository data base for more than 600 children with hereditary and sporadic nephrotic syndrome. In the past two years they have identified two novel genes for hereditary nephrotic syndrome and vesicoureteric reflux (VUR). They have also identified a disease risk allele for childhood onset steroid sensitive nephrotic syndrome (SSNS).
Early life exposure to and colonization with microbes has a profound influence on the education of the immune system and susceptibility to viral and bacterial infections later in life. My research is focused on the influence of the upper respiratory microbiome on the development of recurrent respiratory infections, including acute otitis media (AOM), the leading cause of antibiotic prescriptions and healthcare consultations among children. Importantly, some children develop recurrent infections that are thought to be linked to dysbiosis of the nasopharyngeal microbiome. My overarching goals are to identify alterations in the upper respiratory microbiome associated with AOM and to elucidate host factors and exposures that predispose some children to the development of recurrent AOM episodes.
I have a research interest in the pathogenesis and predictors of progression to Type 2 diabetes in children and adolescents. I examined the relationship between insulin secretion and GAD65 antibody levels at diagnosis on glycemic control in Type 2 Diabetes. I then explored the relationship between body mass index and GAD65 antibody status on β-cell secretion at diabetes onset in African-American children. I subsequently demonstrated that type 2 diabetes in children is associated with abnormalities in cortisol metabolism.
My research is broadly focused on elucidating the complex interactions that exist between the host microbiome and exogenous pathogens that cause infections in children. We have several ongoing projects evaluating: 1) the impact of the upper respiratory microbiome on the risk of colonization and invasion by bacterial respiratory pathogens among infants in Botswana; 2) associations between the gut microbiome of pediatric stem cell transplant recipients and the risk of infections (bloodstream infection, C. difficile infection) and graft-versus-host disease; and 3) the role of the gut and respiratory microbiomes in mediating COVID-19 infection susceptibility and disease severity in children. Ultimately, I aim to develop strategies that use targeted modification of the microbiome for the prevention of infections in children.
A multidisciplinary approach to care of individuals with genetic disorders in conjunction with clinical and bench research that contributes to:
1) An understanding of the natural history and delineation of long term complications of genetic disorders
2) The development of new therapies for genetic disorders through translational research
3) The development and execution of large multicenter trials to confirm safety and efficacy of potential therapies
4) Role of antibodies/immune response in patients on therapeutic proteins.
The focus of our research has been the development of gene therapy with adeno-associated virus (AAV) vectors, most recently by genome editing with CRISPR/Cas9. We have developed gene therapy for inherited disorders of metabolism, especially glycogen storage disease (GSD) and phenylketonuria (PKU).
Dr. Kurtzberg conducts both clinical and laboratory-based translational research efforts, all involving various aspects of normal and malignant hematopoiesis. In the laboratory, her early work focused on studies determining the mechanisms that regulate the choice between the various pathways of differentiation available to the pluripotent hematopoietic stem cell.
I am interested in the genetic and molecular mechanisms of sudden cardiac arrest in children. I am a physician-scientist trained at the confluence of pediatric cardiology, clinical electrophysiology, human genetics, and molecular biology which I apply towards the care for children with heritable arrhythmic disease of the heart. I run a basic science research lab exploring the genetic and molecular causes of sudden cardiac death in children and young adults.
Sarcomas are among the most difficult-to-treat cancers in pediatric oncology, with metastatic forms having the highest mortality. We have established genetically defined human cell-based models and genetically engineered murine models for the pediatric skeletal muscle cancer known as rhabdomyosarcoma. Using these models, we can study the causative role of certain genetic changes (e.g. chromosomal translocations and oncogenic RAS) in rhabdomyosarcoma formation and treatment resistance.
Dr. Mikati’s clinical research has centered on characterization and therapy of pediatric epilepsy and neurology syndromes, describing several new pediatric neurological entities with two carrying his name (POSSUM syndromes # 3708 and 4468), developing novel therapeutic strategies for epilepsy and related disorders particularly Alternating Hemiplegia of Childhood, and applying cutting edge genetic and Magnetic Resonance Imaging techniques to drug resistant pediatric epilepsy.
The primary focus of the laboratory is the study of humoral responses to vaccination and infection with the goal of understanding how to elicit protective responses by vaccination. The laboratory develops antigen-specific reagents for the quantitation and isolation of B cells, and has active projects to study HIV-1, influenza, syphilis, and autoimmunity.
I divide my time between clinic and the laboratory. In clinic I focus primarily on fetal and neonatal patients with genetic disorders, brain malformations, prematurity, perinatal brain injury, and seizures. In the lab, I use single-cell transcriptomic and epigenetic techniques to study cortical neuron development in neurodevelopmental disorders and perinatal brain injury.
My overall research interests are finding effective treatment for human glycogen storage diseases (GSDs) and other inherited metabolic disorders. My current research focuses on identification of novel therapeutic targets and development of effective therapies for GSD II (Pompe disease), GSD III (Cori disease), and GSD IV (Andersen disease) using cellular and animal disease models. The main therapeutic approaches we are using in our pre-clinical studies include protein/enzyme therapy, AAV-mediated gene therapy, and substrate reduction therapy with small molecule drugs.
My research innovatively integrates gnotobiotic murine models, immunology, microbiology, and characterization of the microbiota with the ultimate aim of identifying specific commensal bacteria with immunomodulatory potential and subsequent characterization of their biologic effects. We have recently developed an inventive approach for identifying with high specificity organisms within the microbiota that are causally related to the phenotype of interest.
My research interests include pediatric cardiac intensive care and congenital heart disease.
I am a pediatric oncologist with a clinical and research focus on the care of adolescents and young adults with sarcoma. I have led multiple institutional and national clinical trials exploring new therapies for patients with recurrent sarcoma, and am interested in new drug development and identification of predictive biomarkers.
The immune system protects the host from microbial infection but can cause diseases if not properly controlled. My lab is interested in the receptor signaling mediated regulation of immune cell development and function as well as the pathogenesis and treatment of autoimmune diseases and allergies. We are currently investigating the roles diacylglycerol kinases (DGKs) and TSC1/2-mTOR play in the immune system.
Clinical Research
Children's Clinical Research Unit (CCRU)
Each year, more than 1,000 faculty and study coordinators at Duke enroll patients in clinical trials that involve medical treatment. In 2008, a clinical research organization was created to ensure that these trials are ethically and efficiently managed with the ultimate goal of improving the overall quality of the patient care experience and providing the highest quality clinical research environment possible.
In conjunction with the Duke Office of Clinical Research (DOCR), each academic department or therapeutic area established a Clinical Research Unit to oversee all clinical research requiring IRB approval. The Children's Clinical Research Unit (CCRU) is specific for the Department of Pediatrics.
Duke Clinical Research Institute
The DCRI's Pediatrics faculty ensure that the safety and well-being of children is at the center of every step of pediatric clinical research. Along with statisticians, site and data management experts, and project leaders, the faculty works closely with sponsors to ensure accurate and efficient study delivery.
Clinical Trials
Duke University Medical Center has established a clinical trials network to ensure that patients have access to cutting edge medicines and therapies. Clinical trials take place under the most stringent guidelines, and participants benefit from closely supervised care.
To find current pediatric clinical trials, browse the list or select a category by specialty.
To learn more about participating in clinical trials at Duke, please visit Participating in a Clinical Trial or Study.