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The Children's National Research Institute

Research Programs

We study common pediatric health problems as well as relatively rare inherited diseases. Our strong translational focus includes drug development programs, biomarker discovery, pre-clinical testing, genetic analysis, natural history studies to derive clinical outcome measures, and clinical trials. Below you will find more information regarding specific research areas:

Airway and Lung Diseases

A large team of scientists and investigators at CNRI are researching airway and lung diseases with specific focus on asthma, inflammatory airway diseases, respiratory infections, neonatal and infant airway disorders, human cytomegalovirus, and ciliary dysfunction. 

Bone Health Program

Led by Dr. Laura Tosi, the Bone Health Program seeks to maximize the bone health of children and adults who are at increased risk for fractures by providing multidisciplinary diagnosis and treatment services for them, as well as the opportunity to participate in innovative research.

Brain and Spinal Cord Disorders

Together with scientists in France and Ireland, Dr. Susan Knoblach leads research in the area of central nervous system injury and neurodegeneration disease.  


CNRI investigators and scientists are focusing their research on childhood brain cancers, specifically the diffuse intrinsic pontine glioma (DIPG), which remains one of the deadliest childhood cancers.

Cardiovascular Disorders

The team researching cardiovascular disorders is focusing their efforts on studying heparin-binding growth factors. 

Gender-Based Biology

Read more about the gender-based biology research happening at CNRI and how investigators are exploring the etiology of disorders/differences of sex development and neural stem cells and brain sex differences.

Rare Disease Institute

Dr. Marshall Summar and his team are working together across a number of projects in a first-of-its-kind center focused exclusively on advancing the care and treatment of children and adults with rare genetic diseases.

Steroid Drug Development and Inflammatory Bowel Diseases

Investigators and researchers in this program are studying inflammatory bowel disease and are working on developing dissociative steroid drugs to improve the efficacy and decrease the side effects associated with glucocorticoid drugs.

Urea Cycle Disorders (UCD)

The Children’s National Research Institute is considered the world leader in the diagnosis, treatment, and research of urea cycle disorders with three renowned experts leading the research in this field.