UPTiC Mentors

Andrew Landstrom, MD, PhD, (Track Lead, Duke) focuses on identifying the genetic and cellular causes of heritable cardiac diseases including arrhythmia syndromes (cardiac channelopathies) and primary disease of the myocardium (cardiomyopathies) to determine the mechanisms of these diseases so that at-risk individuals can be identified and new, personalized, therapies can be developed.

Misty Good, MD, (Track Lead, UNC) studies the cellular and molecular mechanisms underlying the pathogenesis of necrotizing enterocolitis as well as signaling pathways that mediate the dysregulated immune response to determine how these responses can be prevented through targeted therapies.

Eric Benner, MD, PhD, (Duke) studies the molecular mechanisms of oxysterol-induced oligodendrogenesis in the context of neonatal brain injury and adult demyelinating diseases such as multiple sclerosis, with the intention to translate these oxysterols into a potential therapy for diffuse white matter injury.

Toni Darville, MD, (UNC) studies the relationship between Chlamydial infection and immune responsiveness in adolescent girls and young women, with important implications for vaccine development.

Ian Davis, MD, PhD, (UNC) studies the relationship between chromatin and transcriptional regulation in cancer and development, using chromatin-based strategies, genomic approaches and genome-wide computational analytics for fundamental mechanistic studies, small molecule screening and drug discovery, and for the development of novel diagnostics.

Mai Elmallah, MD, (Duke) focuses on the control of breathing, the impact of genetic disorders on respiratory physiology, and novel therapeutics to treat respiratory insufficiency in neuromuscular diseases.

Erin Heinzen, Pharm D, PhD, (UNC) leads a research program focusing on the genetics of neurodevelopmental diseases, which has contributed to the discovery of 15 novel epilepsy genes, and other neurological disorders.

Matthew Hirschey, PhD, (Duke) studies metabolic control, mitochondrial signaling, and cellular processes regulating human health and disease, including diabetes, cardiovascular disease, and cancer, with the goal of exploiting metabolic regulation for its therapeutic potential.

Matthew Kelly, MD, MPH, (Duke) develops microbiome-based therapies for the prevention and treatment of infections, particularly those that have a higher burden among the world’s poorest children.

Nancie MacIver, MD, PhD, (UNC) studies nutritional immunology and immunometabolism, with attention to how pharmacological modulation of metabolism, nutritional status and nutritionally-regulated hormones affect T cell immunity in the context of health and disease.

Tony Moody, MD, (Duke) develops new tools to study B cell responses to vaccines and infection, including HIV-1, influenza, and syphilis maintenance and manages a large biorepository at the Duke Human Vaccine Institute of samples for human clinical trials, human observational studies, and non-human primate preclinical studies.

Chengwen Li, PhD, (UNC) explores novel strategies for development of adeno-associated viral (AAV) vectors with the ability to target human tissues, with a current focus on mitigating the cytotoxic T cells response to AAV vectors and optimizing AAV vectors for hemophilia gene therapy.

Corinne Linardic, MD, PhD (Duke) uses models of rhabdomyosarcoma to probe the molecular contributions of signature mutations, including oncogenic RAS and PAX3::FOXO1, to the developmental pathways leading to treatment resistance and relapse, with the goal of developing novel cancer therapeutics for children.

Kenneth Pearce, PhD, (UNC) conducts early drug and chemical probe discovery research, including development of biochemical and cell assays and mechanistic/biophysical studies to approach a wide variety of therapeutic areas including antibacterials, antivirals, metabolic diseases, cancer, inflammatory diseases, and tissue fibrosis.

Barbara Savoldo, MD, PhD, (UNC) developments new vectors, targets, and clinical applications for chimeric tumor specific T-cell-directed therapeutics (CAR-T cells) and directs the GMP Advanced Cellular Therapies facility that generates modified T-cells for first-in-human and first-in-child use.

Mari Shinohara, PhD, (Duke) studies the basic signaling mechanisms in receptor mediated innate immunity in the context of multiple sclerosis, Alzheimer’s disease, and infections, providing an important foundation for therapeutic intervention.

George Truskey, PhD, (Duke) studies the response of cells to physical forces, cardiovascular and skeletal muscle tissue engineering, and microphysiological systems for disease modeling and drug and toxicity testing.

Mark Zylka, PhD, (UNC) conducts neuroscience research focused on nociception, pain modulation, and autism, including G protein-coupled receptors in nociceptive.

Dwight Koeberl, MD, PhD, (Track Lead, Duke), develops new therapies for glycogen storage diseases using animal models and translates these discoveries into clinical trials; he has used gene therapy to reveal a novel therapeutic target, leading to completion of a Phase I/II study of a β2-agonist drug, clenbuterol, in late-onset Pompe disease.

Michelle Hernandez, MD, (Track Lead, UNC), studies respiratory and systemic innate immune response in inhalation injury, respiratory immunology and inflammatory lung diseases, including clinical trials of agents to inhibit inflammation to mitigate disease severity and the application of novel therapeutics for difficult-to-control asthma.

Michael Cohen-Wolkowiez, MD, PhD, (Duke) is member of the DCRIwith a highly successful career in pediatric clinical trials, clinical pharmacology and drug development, including developing novel clinical trial methods to  support feasible studies in children and infants.

Stephanie Davis, MD, (UNC) studies the pathogenesis of respiratory diseases affecting young children with cystic fibrosis, primary ciliary dyskinesia, and respiratory morbidity associated with preterm birth in order to develop novel predictive and therapeutic strategies.

Evan Dellon, MD, MPH, (UNC) focuses on optimizing the diagnosis, characterizing the epidemiology, studying the pathogenesis, and refining the treatment and monitoring of eosinophilic esophagitis and other eosinophilic gastrointestinal disorders, with the overall goal of improving patient care and outcomes.

Rachel Greenberg, MD, MS, MHSCR, (Duke) leads clinical trials and epidemiology studies aimed to improve safety and efficacy of drugs in infants and children.

Kevin Hill, MD, MS, (Duke) focuses on improving outcomes in children with heart disease with particular interest in early and late phase clinical drug trials.

Christoph Hornik, MD, PhD, MPH, (Duke) develops novel and efficient methods to study health interventions, including pharmaceuticals and risk factor modifications, through traditional prospective multi-center trials, real world databases, and innovative study designs including regulatory grade research registries and direct-toparticipant studies.

Corinne Keet, MD, PhD, (UNC) studies the prevention and treatment of allergic diseases in childhood by leading a large multi-center cohort trial focused on understanding the pathogenesis of food allergy and atopic dermatitis in the first years of life, as well as through leadership of other clinical studies of childhood allergic diseases.

Priya Kishnani, MD, MBBS, (Duke) performs translational and clinical research for the treatment of inborn errors of metabolism, especially lysosomal storage diseases and glycogen storage diseases, including Pompe disease; as Director of the Chen Center for Pediatric Genetics and Genomics she leads efforts to develop new therapies for rare genetic disorders.

Matt Laughon, MD, MPH, (UNC) leads research on neonatal epidemiology, pharmaco-epidemiology, and clinical pharmacology and is an expert in the execution of clinical trials of therapeutics in infants with a track record of successful inter-institutional mentorship.

Joseph Muenzer, MD, PhD (UNC) is an internationally regarded expert on the diagnosis, management, and clinical treatment of patients with inborn errors of metabolism, especially mucopolysaccharidoses, with extensive expertise in study design and implementations for clinical trials developing intravenous enzyme replacement therapy.

Jennifer Li, MD, MHS, (Track Lead, Duke), focuses on pediatric clinical trials and outcomes studies involving the cardiovascular system, leading efforts in drug development to improve the dosing, safety, and efficacy of pediatric therapeutics and to develop study methods for pediatric outcomes with unique regulatory experience of translating research findings into medical practice.

Michael D. Kappelman, MD, MPH, (Track Lead, UNC) is a pediatric gastroenterologist and health services researcher focused on clinical and comparativeness effectiveness research in chronic conditions including Inflammatory Bowel Disease.

Sarah Armstrong, MD, (Duke) is devoted to improving the treatment of children and adolescents with obesity, particularly those from underserved backgrounds who experience social, ecological, and environmental barriers to health, using quantitative, qualitative, and community-engaged research methods.

Sylvia Becker-Dreps, MD, MPH, (UNC) studies viral gastroenteritis, Zika virus infection, and the evaluation of vaccine effectiveness in both the US and in low- and middle-income countries.

Scott Commins, MD, PhD, (UNC) studies the processes that control IgE antibody production and allergic reactions to food, with a special focus on discovering the immunologic mechanisms leading to alpha-gal red meat allergy,as well as its epidemiology, to inform ongoing research in humans to design effective therapies.

Thomas Ferkol, MD, (UNC) is a pulmonologist with clinical and research interests in defining the genetics, pathogenesis, and clinical manifestations of inherited, suppurative lung diseases, including cystic fibrosis,primary ciliary dyskinesia, and non-cystic fibrosis bronchiectasis, with the goal of informing novel therapeutic development for these pediatric diseases.

Emily O’Brien, PhD, (Duke) is a health services researcher and epidemiologist focused on using real-world data to evaluate comparative effectiveness of commonly used therapies for patient-prioritized outcomes in cardiovascular disease and has expertise in observational studies and pragmatic clinical trials, working with multiple data sources, including linked data from registries, patient self-report, epidemiologic cohorts, electronic health records, and administrative claims.

T. Michael O’Shea, MD, MPH, (UNC) leads epidemiologic studies of brain damage and neurodevelopmental outcome in low gestational age newborns and developmental follow up of participants in neonatal clinical trials, as well as tracking environmental exposures in utero and early childhood as an underlying cause of disease in childhood including epilepsy, autism, and ADHD.

Bryce Reeve, PhD, (Duke) focuses on enhancing patient-reported outcomes in clinical research and improving the quality of care for pediatric and adult cancer patients, including the development of qualitative and quantitative measures and integration of patient-reported outcome data in research, including clinical drug trials, and healthcare delivery.

Matthew Raubach, MD, (Duke) is a global health Medicine-Pediatrics trained infectious disease clinical and epidemiological researcher focused on improving the recognition, diagnosis, and treatment of severe febrile illness in resource-limited tropical settings.

Betsy Sleath, PhD, (UNC) focuses on provider-patient communication about medications and how this impacts adherence and patient-centered outcomes in childhood and adolescence, recruiting patients from diverse socioeconomic, ethnic, and racial backgrounds into clinical research studies.

P. Brian Smith, MD, MPH, MHS, (Duke) is an expert in clinical trial design, clinical research, and biostatistics, focusing his career on pediatric pharmacology, drug safety, and outcomes research. He is currently PI for the Coordinating Center for the Environmental Influences on Child Health Outcomes.

Til Stürmer, MD, PhD, (UNC) directs the Center for Pharmacoepidemiology, leads a pharmacoepidemiology training program, is a member of the FDA Drug Safety and Risk Management Advisory Committee, and conduct research on methods of nonexperimental treatment comparisons, including comparative effectiveness.

Kanecia Zimmerman, MD, MPH, (Duke) focuses on provider-patient communication about medications and how this impacts adherence and patient-centered outcomes in childhood and adolescence, recruiting patients from diverse socioeconomic, ethnic, and racial backgrounds into clinical research studies.