Stem Cell Program

Stem Cell Program

Every day, the medical staff and researchers at Boston Children's Hospital witness the devastating effects of diseases like leukemia, diabetes, sickle cell anemia and heart disease on the lives of the children they treat. Every day, we see very small patients fighting very large battles just to stay alive. We see toddlers spending their days in blood transfusion units instead of on playgrounds, and teenagers living with a restrictive regimen of treatments and the knowledge that their promise of a future is uncertain. For them, and for their families, the hope is very simple: Please find a cure for our disease.

Our patients’ health and a future filled with promise are what drive the researchers, faculty and staff of the Stem Cell Program at Boston Children’s Hospital.

15 years of stem cell research

Leonard Zon, M.D. and George Q. Daley, M.D., PhD launched the Stem Cell Program at Boston Children’s Hospital in 2004. 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.

Our mission

This program’s sole mission is to explore, understand, and translate the promise of stem cells into effective clinical therapies and treatments. Its leaders have developed a three-pronged strategy to fulfill this charge:

  1. Develop basic methods for creating customized stem cells that can be turned into any tissue in the body.
  2. Apply those methods to the diseases most likely to yield the first breakthrough treatments. Blood diseases such as leukemia are likely to be first because the science is furthest along for them and the method for delivering the cells, blood stem cell transplantation, is well established. These disorders are the Program’s primary focus, but major progress in its iPS cell research has enabled expansion to other diseases more rapidly than anticipated. Intensive research now focuses on solid cancers, liver diseases, and rare conditions that shed light on more common ones.
  3. Make iPS cells and other technologies developed here available to colleagues worldwide and so speed progress on a vast array of disorders.

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.

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.



Equity and inclusion

The Stem Cell Program at Boston Children’s Hospital is committed to upholding Boston Children’s Declaration on Equity, Diversity, and Inclusivity. We are committed to encouraging and growing the program’s diversity and inclusion across race, gender, age, religion, identity and experience within our program. We are steadfast in building our diversity and cultivating researchers and scientists who advocate for our mission and are models to the hospital and research community.

Stem Cell Research

Boston Children's Hospital Stem Cell Program @BCHStemCell 5/7/2021 3:02:11 PM
Check out our open positions @BCHStemCell: Research Administrator II Find out more about joining the Stem Cell…
Boston Children's Hospital Stem Cell Program @BCHStemCell 5/6/2021 4:52:40 PM
RT @nansonaham: A mechanism that controls the 'dosage' of #miRNAs, thereby controlling embryonic germ layer specification – kudos to Richar…
Boston Children's Hospital Stem Cell Program @BCHStemCell 5/6/2021 3:01:18 PM
Congratulations to @ireneGLwong for receiving the 2021 Student Leadership Award for outstanding service as a BBS Pe…
Boston Children's Hospital Stem Cell Program @BCHStemCell 5/6/2021 12:00:03 PM
RT @BCH_Innovation: Controlling embryonic germ layer specification by controlling the “dosage” of miRNAs – intricate work led by Richard Gr…
Boston Children's Hospital Stem Cell Program @BCHStemCell 5/5/2021 8:35:52 PM
RT @BostonChildrens: Health experts are hopeful that the @pfizer #COVID19 vaccine may soon be authorized for use in youth age 12-15. @rickm…
Boston Children's Hospital Stem Cell Program @BCHStemCell 5/5/2021 7:03:52 PM
RT @mehdi_pirouz: Check out the recent paper from Gregory lab published today @nature. Happy to be a part of the story.…
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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