Our Research

Translating innovative research into outstanding patient care.

A New Era of Research

Our research teams solve the most challenging problems in children’s health. How do we make these breakthroughs? Our scientific community thrives on collaboration from bench to bedside that leads to effective therapies, medical devices, and treatment techniques. When we have an important pediatric research question to answer, our lab investigators produce novel understanding about basic life processes, and our physician-scientists use these insights to inform patients’ clinical experiences. We align that expertise with our first-of-its-kind multidimensional data collection. The result is unparalleled knowledge creation. From laboratory discoveries like personalized cellular immunotherapy treating childhood cancer, to the development of new vaccines preventing disease globally, to establishing public polices improving children’s safety, our work is transforming our patients’ and their families’ lives.

Our Major Scientific Themes

We ask novel research questions that bridge disciplines in targeted scientific areas and span the research continuum to accelerate scientific discoveries that improve the health of children.

Rare & Complex
Fetal to Adult
Novel Therapeutics
Precision Health

Rare and Complex Diseases

We invest in basic science capabilities, biobanking, and clinical and translational expertise. Our robust capabilities allow us to synthesize the wealth of information in our research databases to streamline scientific efforts toward rapid and accurate diagnosis, new therapeutic interventions, and enhanced quality of life for children living with a rare disease or complex condition.

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DNA Vector Art
Our Major Scientific Themes

Fetal to Adult Continuum

We are uniquely positioned with the University Pennsylvania to provide a high-performing infrastructure supporting research across the fetal-adult continuum. World-class research tools and integration and analysis of data streams from multiple sources allows us to tackle critical questions about common, complex, and chronic diseases such as heart disease, cancers, diabetes, and mental illnesses.

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Fetal to Adult
Fetal to Adult Continuum

Novel Therapeutics and Devices

We transform discoveries into new drugs, therapies, software, medical devices, and mobile apps for pediatric medical conditions. Prominent examples include pioneering work in fetal surgery, gene therapy for hereditary disorders, and application of cellular immunotherapy for children with highly refractory cancers. Our culture of innovation helps them turn early stage ideas into reality.

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Novel Therapeutics and Devices
Novel Therapeutics and Devices

Precision Health

We get the right preventive or therapeutic intervention to the right child in the right place at the right time. Our precision health research breakthroughs lead the way to personalized medicine that provides more meaningful diagnostic results, tailors treatments to children’s genetic profiles, and customizes care to meet our patients’ needs at all developmental stages.

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Precision Health
Precision Health
Cornerstone Blog
Take a multimedia look at our HIV researchers' work – from our basic science labs, to our Center for Functional Neuroimaging, to Botswana's neighborhoods.
Cornerstone Blog
Take a tour of the Aquatic Zebrafish Core on Cornerstone, and learn all about these marvelous underwater superheroes who help scientists save lives.