Stem Cell Program

Extraordinary potential for therapies that may change the future.

Stem Cell research holds great promise for advances in science, medicine, and in the lives of children and adults living with serious and diseases. The physician-scientists and researchers at Boston Children's Hospital believe stem cell biology holds the key to treatments for a wide range of complex conditions.

What are Stem Cells?

Stem cells are single cells with two unique qualities: they can make endless copies of themselves, and they can mature into a variety of specialized cells. These qualities make stem cells promising tools in medicine, allowing patients to receive needed cells or tissues, or have diseased cells or tissues replaced with healthy ones. Grown in the lab, genetically repaired if needed, and coaxed to become a specific tissue, stem cells allow doctors to patch a scarred heart, reawaken damaged nerves or reboot an immune system incapable of fighting infection. Stem cells are invaluable to scientists in understanding human disease.

Researchers & clinicians at Boston Children’s Hospital believe stem cell research holds extraordinary promise for the children we treat and for countless others around the world. The mission of the Stem Cell Program at Boston Children’s is to explore, understand, and translate this promise into clinical therapies and treatments.

What types of stem cells are being studied at Boston Children’s?

 Pluripotent stem cells

Able to make cells from all basic body layers — can produce any cell or tissue the body needs to repair itself.



 Adult stem cells

Found in infants or adults — specialized stem cells that give rise to one or more specific cells or tissues.


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Explore our research

Blood Disorders and Stem Cells

Doctors at Children's Hospital Boston currently use bone marrow stem cells to treat a variety of blood diseases, including leukemia and different anemias.


Stem cells help doctors at Children's Hospital Boston understand the nature of cancer and how it may be treated. This includes research on "cancer stem cells," the wayward stem cells that allow cancers to grow and survive.

Muscular Disorders

In addition to disease modeling, scientists are interested in how stem cells, particularly muscle stem cells, can treat muscular dystrophy directly.

Heart Disease

Research from Children's Hospital Boston on heart stem cells may lead to therapies in older people with heart conditions, such as heart attack survivors.

Congenital and Genetic Disorders

Stem cell research at Children's Hospital Boston can help doctors find new approaches to treating the congenital and genetic disorders that affect our youngest and most vulnerable patients.

Neurologic Disorders

Stem cell research at Children's Hospital Boston can help doctors understand, and possibly treat, the neurological disorders that may affect anyone, from newborns to senior citizens.
Building on our extraordinary leadership in pediatric science at Boston Children’s, we translate our findings — and those of others — to fundamentally change how disease is treated and cured.

David A. Williams, MD
Senior Vice President and Chief Scientific Officer

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