About Research
Breakthrough Discoveries

A Leader in Scientific Discovery and Innovation

With more than $304.7M in external funding and 4,729 publications in 2023, Cincinnati Children's serves as one of the nation's most significant sources of pediatric scientific discovery and innovation.

Our scientists work along the full continuum of research with an unrelenting focus on curing childhood diseases. From Albert Sabin’s oral polio vaccine to the identification of lung surfactant proteins, we’ve been contributing to life-changing and life-saving discoveries for more than 90 years. Learn more about our breakthrough discoveries.

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2023

Basic Science: Chasing the Undiscovered
From secrets of brain development to mysteries of somite segmentation, scientists at Cincinnati Children’s pursue deeper understanding of the human condition.
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Preclinical Science: Building the Platforms of Discovery
From developing advanced organoids to inventing ways to deliver therapy to muscle tissues, Cincinnati Children’s researchers are leaders in creating new tools that can accelerate the chase for cures.
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Clinical Science: The Art of Moving Discovery from Bench to Bedside
Be it carrying out classic three-phase clinical drug trials or following other closely regulated avenues of human subjects research, investigators at Cincinnati Children’s are leaders in translating lab breakthroughs into real treatments that improve outcomes.
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Clinical Implementation: Accelerating Best Practices
From advancing the use of virtual reality training to preventing cardiac arrest during intensive care, clinician-scientists at Cincinnati Children’s strive to be the best at getting better.
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Population Health and Equity: A Quest to Improve Outcomes for All Children
Be it addressing inequities in hospital care, remaining ever-vigilant about disease outbreaks, or applying research expertise in new ways to achieve population scale impact, science at Cincinnati Children’s extends well beyond our walls.
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2022

‘Super’ Antibody Discovery Opens Doors for New Vaccines, Therapies
An eye-opening study, published in Nature, reveals how the early days of mother-to-newborn immunity actually work. The findings suggest that researchers may be able to mimic amped-up antibodies produced by expecting mothers to create new drugs and vaccines to prevent a wide range of infectious diseases.
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Novel Approach Generates Most Complex Stomach, Esophagus Organoids to Date
Using a new, three-layered assembly approach, scientists at Cincinnati Children’s report success at developing a stomach organoid so sophisticated that it has distinct glands and nerve cells that can control smooth muscle contractions. The study, published in Cell Stem Cell, reflects the latest in a string of organoid medicine breakthroughs achieved at Cincinnati Children’s.
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In Uganda, Rheumatic Heart Disease Research Reaches its GOAL
While rare in the United States, more than 40.5 million people globally are living with rheumatic heart disease, and more than 306,000 people die each year from a condition that starts from seemingly minor strep throat infections. Now, results from the GOAL trial in Uganda, published in the New England Journal of Medicine, show that low-cost penicillin treatments can sharply reduce latent rheumatic heart disease progression.
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Two-Hit Model of Muscular Dystrophy May Redirect Hunt for Cures
For most people, muscle injury healing is spurred along by muscle satellite cells that function to regenerate the tissue. But for people with muscular dystrophy, those satellite cells actually contribute to muscle wasting, according to findings published in Nature Communications.
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Targeting RipIL-33 Pathway Could Transform Allergy Treatment
The unexpected discovery of a built-in rapid reaction system that triggers inflammatory responses when people are exposed to allergens from insects, mites and fungi, may solve a mystery that has puzzled immunologists for years. The result could be a new wave of improved treatments to stop allergic inflammatory reactions, according to findings published in Nature Immunology.
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Five Childhood Risk Factors Predict Risk of Adult Cardiovascular Events
Cincinnati Children’s scientists played central roles in a massive international study that tracked more than 38,000 people across decades to measure how closely childhood signs of heart disease risk predicted actual adult heart conditions. They found five risk factors that count, and report that taking early steps to address those risks can pay off later in life. Findings were published in The New England Journal of Medicine.
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2021

Pfizer-BioNTech COVID-19 Vaccine Safe and Effective for Adolescents
This report on the effectiveness of the Pfizer-BioNTech vaccine helped launch the wave of emergency approvals that made COVID-19 vaccines available to younger groups of children.
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‘Massively Parallel’ Gene Screening Tool Can Accelerate Research for Nearly Any Disease
This hunt for deeper understanding of the causes of lupus, a chronically painful disease that can cause severe organ damage, led to an innovative gene screening tool that can accelerate research for nearly any disease.
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How Clock Genes Drive Healthy Spinal Formation
A study exploring spine development in zebrafish reveals the crucial role that well-synced internal clocks play in early development, with long-range implications for understanding and preventing human birth malformations.
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Bone Marrow ‘Map’ Opens Path to Organoid-like Blood Stem Cell Production
An unprecedented “map” that details the mechanisms at work in bone marrow formation may someday help clinicians replace cancerous bone marrow cells with healthy, patient-specific marrow cells grown in the laboratory.
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Sunlight Reaches Deep into Brain to Play Key Role in Metabolism
This discovery—that specific wavelengths of natural sunlight actually pierce the skull to reach light-sensing cells deep inside the brain—has numerous implications for how to manage metabolism-related disorder like Type 2 diabetes and other conditions.
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Gene Variants Increase Risk for Dysfunctional Skin Barrier, Asthma, and Food Allergies
Discovering two gene variants among children who develop severe allergies after developing atopic dermatitis (eczema) adds evidence to a rising theory that skin health is more closely connected to lung and gut health than many have suspected.
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Tissue Healing in the Gut Depends on Help from Friendly Bacteria
Meanwhile, another study conducted here further illustrates how much human health can depend upon the “good” bacteria living inside us. Damaged intestines need help from bacteria to heal.
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2020

Single-Cell Approach Reveals Impact of Disease-Causing Gene Mutations
Kyle Ferchen, BS, H. Leighton Grimes, PhD, Nathan Salomonis, PhD, and Andre Olsson, PhD
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World's First Three-Organoid System Opens Doors for Medical Research Diagnosis
Kentaro Iwasawa, MD, Jim Wells, PhD, Aaron Zorn, PhD, and Takanori Takebe, MD, PhD
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Dose Escalation Sharply Improves Hydroxyurea Benefit for Children with Sickle Cell Anemia
Russell Ware, MD, PhD, Teresa Latham, MA, and Adam Lane, PhD
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Clinical Trial Success for HLH Treatment
Alexi Grom, MD, and Michael Jordan, MD
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Cardiac Stem Cell Therapy Improves Scar Formation Rather Than Prompting Cardiomyocyte Growth
Ronald Vagnozzi, PhD, and Jeffery Molkentin, PhD
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2019

First Human Esophagus Organoid Grown from Pluripotent Stem Cells
James Wells, PhD and Stephen Trisno
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Probiotics Show No Benefit for Children with Acute Gastroenteritis
David Schnadower, MD
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PROTECT Study Produces New Model for Treating Children with Ulcerative Colitis
Lee Denson, MD
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Daily Hydroxyurea Pill Safely Lowers Sickle Cell Burden Among African Children
Russell Ware, MD, PhD, Leon Tshilolo, MD, PhD, Adam Lane, PhD, Susan Stuber, MA, Teresa Latham, MA and Patrick McGann, MD
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CYCLOPS Algorithm Helps Build Database of Cycling Genes in 13 Human Tissues
Marc Ruben, PhD, John Hogenesch, PhD, and David Smith, MD
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How the Gut Microbiome Influences Lean Vs. Obese Phenotypes
Theresa Alenghat, VMD, PhD, Jordan Whitt, Vivienne Woo, Patrick Lee, Jessica Moncivaiz, Yael Haberman Ziv, MD, PhD, Lee Denson, MD, and Patrick Tso, PhD
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How Light Therapy Might Prevent Vision Problems in Preterm Infants
Minh-Thanh Nguyen, PhD, and Richard Lang, PhD
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2018

Epstein-Barr Virus Linked to Seven Serious Diseases
Matthew Weirauch, PhD, Leah Kottyan, PhD and John Harley, MD, PhD
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Nerve Pruning in Mice Sheds Light on Human Fine Motor Control
Yutaka Yoshida, PhD
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Cracking the Genetic Code of Malignant Peripheral Nerve Sheath Tumors
Q. Richard Lu, PhD and Lai Man Wu, PhD
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Hepatocyte Transdifferentiation: How Some Liver Cells Switch Identities to Build Missing Bile Ducts
Stacey Huppert, PhD
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Hospital, Clinics, Schools Team Up to Improve Asthma Outcomes
Andrew Beck, MD, MPH, Carolyn Kercsmar, MD and Mona Mansour, MD, MS
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Six Gene Loci Associated with Gestational Duration and Spontaneous Preterm Birth
Louis Muglia, MD, PhD and Ge Zhang, MD, PhD
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Thermoneutral Mouse Model Opens Doors for NAFLD Research…and Beyond
Senad Divanovic, PhD
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2017

AERSMine Helps Investigators Dig Into Massive FDA Database
Bruce Aronow, PhD, Mayur Sarangdhar, PhD and Anil Jegga, DVM, MRes
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Stomach Organoid Success Advances Work to Build a Complete GI Tract on a Chip
James Wells, PhD and Kyle McCracken, MD, PhD
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TRAF6 Role Extends Beyond MDS to Affect Innate Immune Signaling
Daniel Starczynowski, PhD
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Targeting C5ar1 Shows Early Promise as New Therapy for Gaucher Disease
Manoj Pandey, PhD
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Bold Findings About Blood Cell Formation Stir the Scientific Pot
H. Leighton Grimes, PhD, Harinder Singh, PhD and Nathan Salomonis, PhD
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Better AWAREness of Acute Kidney Injury Could Transform Intensive Care
Stuart Goldstein, MD
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CHAMP Study Challenges Medication-Driven Approach to Migraine Care
Andrew Hershey, MD, PhD and Scott Powers, PhD
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Inhibiting MEK Protein Shrinks NF1 Plexiform Tumors
Nancy Ratner, PhD, Brian Weiss, MD and Jianqiang Wu, MD
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Scientists Tissue-Engineer Human Intestines with Functioning Nerves
Michael Helmrath, MD, James Wells, PhD and Samantha Brugmann, PhD
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2016

New SpDamID Tool Opens New Doors
Matthew Hass, PhD and Raphael Kopan, PhD
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Sickle Cell Breakthrough: A Pill to Replace Blood Transfusions
Russell Ware, MD, PhD
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Breakthrough Therapy Improves Life for Children with LRBA Deficiency
Michael Jordan, MD
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A New Path to Helping Hearts Heal Themselves
Katherine Yutzey, PhD
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Six Variables Explain Wide Racial Disparity in Asthma Readmission Rates
Robert Kahn, MD, MS, Andrew Beck, MD, MPH and Bin Huang, PhD
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Bariatric Surgery Improves Outcomes for Teens in Weight, Heart Health and Quality of Life
Stavra Xanthakos, MD, MS, Thomas Inge, MD, PhD and Michael Helmrath, MD, MS
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2010-2015

The Development of Intestine and Stomach Organoids
James Wells, PhD
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Changing the Direction of Cardiac Stem Cell Research
Jeffery Molkentin, PhD
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Gene Therapy Advances Raise Hopes for Curing Sickle Cell Disease
Punam Malik, MD
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Identifying and Understanding Eosinophilic Gastrointestinal Disorders EGID
Marc Rothenberg, MD, PhD
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Macrophage Transplantation May Cure Pulmonary Alveolar Proteinosis Pap
Bruce Trapnell, MS, MD
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Developing Pediatric Learning Health Systems
Peter Margolis, MD, PhD
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2000s

Researchers demonstrate that divalproex sodium (Depakote) is effective in significantly improving symptoms of bipolar I disorder in children and adolescents.
Robert B. Hinton, Jr., MD, and colleagues are the first to show the high heritability and likely genetic underpinnings of Hypoplastic Left Heart Syndrome (HLHS), a rare but deadly heart malformation.
In the first nationwide Attention Deficit Hyperactivity Disorder (ADHD) study, researchers find that approximately 2.4 million children meet ADHD diagnostic criteria, yet more than half are undiagnosed.
Researchers discover that prenatal exposure to tobacco smoke and childhood exposure to lead account for more than one-third of Attention Deficit Hyperactivity Disorder (ADHD) cases among children in the United States.
The crucial role of the protein RhoH GTPase in the development and activation of cells critical to the immune system is identified. The findings, along with other studies, suggest that RhoH GTPase may provide a target for therapeutic intervention in some types of leukemia.
The first gene associated with eosinophilic esophagitis is discovered.
European officials approve a rotavirus vaccine developed and tested by two Children's researchers. The decision by the European Commission makes Rotarix the first rotavirus vaccine available to children throughout Europe.
Creatine transporter deficiency is identified, which can explain developmental delays in some male children.
Revealed the genetic underpinning to biliary atresia, the most common reason for liver transplant in children.
Developed a genital herpes vaccine that was 75 percent effective in female patients during trials and may help prevent the spread of the virus from mother to newborn.
A genetically engineered vaccine is the first to be proved effective in protecting newborns, in an animal model, against cytomegalovirus (CMV), the most common congenital viral infection in the United States.
Exposure to environmental tobacco smoke, even at extremely low levels, is associated with decreases in certain cognitive skills, including reading, math and logic, and reasoning in children and adolescents.
The first study to show adverse consequences of blood lead levels below 10 micrograms per deciliter, the level that is currently considered 'acceptable.'
Development of a new vaccine against rotavirus, a leading cause of death in infants and children worldwide.

1990s

Discovery of a gene and identification of a key protein involved in the development of allergic diseases.
Findings that parental obesity more than doubles a child's risk of obesity in adulthood.
Discovery of flavohemoglobin's role in protecting bacteria from the body's immune system. May lead to a new generation of more effective antibiotics.
Documentation that ATP depletion leading to end organ failure in shock can be pharmacologically delayed. Could improve the chances of surviving gunshots, car accidents and massive infections.
Demonstration that immunization can be effective for treating, as well as preventing, genital herpes virus infection; and creation of a compound that appears to provide total protection against herpes virus transmission.
Discovery that estrogen is an essential regulator of male adolescent growth and bone mineralization.
Discovery of multiple enzyme defects in bile acid synthesis that cause liver failure -- now treatable.

1940s-1980s

Identification and cloning of surfactant proteins, important for optimal surfactant replacement therapy. The introduction of artificial surfactant, used nationwide since 1989 to help improve lung function in premature babies, saves an estimated 2,000 lives a year in the United States.
Identification of unique morphological features of Reye syndrome.
Development of the Sabin oral polio vaccine, which has conquered polio in the western hemisphere.
Development of the first artificial lung compounds (perfluorocarbons), now also used for assisted ventilation.
Creation of the first heart-lung machine, opening the door for modern heart surgery.
Seminal studies indicating the effects of environmental factors on prenatal development.

Black and white photo of a doctor holding up a test tube.

Historic Breakthroughs

Cincinnati Children’s has a long history of innovation, advancing pediatric care since 1884.

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