The physician-scientists and researchers at Boston Children’s Hospital 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 and Schlaeger labs have created more than 100 disease-specific hiPS 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.
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.
Pioneering new technologies
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Barcoding leaves its marks on cells
Our researchers are pioneering a groundbreaking technology—barcoding—in which they "fingerprint" each stem cell with a unique DNA sequence. Because each barcode gets passed on to all the stem cell's progeny, researchers can use it to trace each cell's lineage and study how some blood cancers, such as leukemia, may arise.
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Exploring RNA biology
By the time the COVID-19 vaccine thrust RNA into the limelight, scientists were well aware of its potential. Sometimes chemicals such as methyl get added to RNA's surface, giving it the power to boost protein production. But if too much is added, our health can suffer. Two of our investigators are studying these modified RNAs to help understand diseases and to guide treatment development.