Research on Diseases

The physician-scientists and researchers at Children’s Hospital Boston believe that stem cell biology holds the key to treatments for a wide range of currently untreatable or incurable diseases. Much of our current work centers on specific diseases and the ways in which stem cells might be used to model and understand those diseases. Critical work is also underway to explore how the power and nature of stem cells might be harnessed in the development of general and patient-specific therapies.

To achieve this, we are intensively exploring all pathways available—including embryonic stem cells, induced pluripotent cells (iPS cells) and adult stem cells. By engaging on multiple fronts, we increase our ability and potential to unlock the door to treatments for many diseases. Already, clinical trials are underway on a drug discovered by the Zon Lab that has the potential to boost production of blood stem cells, with significant implications for the treatment of patients with leukemia. The Daley Lab has created more than 20 disease-specific iPS cell lines that will enable researchers to track the origins of a specific disease and to attempt to change its course. Many other exciting investigations are underway and are discussed in these pages.

Take a virtual tour through the Daley Lab to learn more:

Our team of scientists are exploring ways to understand and treat blood, neurological, kidney, lung and heart disease; cancer and diabetes; disorders of the muscular and immune systems; and congenital and genetic disorders. Every day brings the potential for new insights, new discoveries, and new hope that the vast promise of stem cells can be realized, and that people suffering from these diseases — both children and adults — can be cured. We are committed to the realization of that goal.

To date, there are more than a dozen diseases represented in the Stem Cell Program’s research and the program is constantly adding new diseases to its research roster. Visit this page and our newsroom often for updates on our research.

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