The research enterprise at Boston Children’s Hospital, comprising more than 3,000 researchers, is the world’s largest at a pediatric center. Our work is fueled by a deep understanding of disease coupled with world-class discovery platforms, including genetics and genomics, gene editing, bioinformatics, proteomics, bioengineering, image analysis, multiple animal models, biobanks and disease-specific stem cell lines. We have special expertise in rare disease discovery and see large, highly diverse patient populations.

From ADHD to Zika virus, we partner closely with scientists worldwide. To accelerate translation of new therapies and technologies to the bedside, we partner with industry through such programs as the Technology and Innovation Development Office (TIDO), the Innovation & Digital Health Accelerator (IDHA) and the Translational Neuroscience Center.

Image of a mouse brain that received a direct transplantation of hematopoietic stem cells.

Gene therapy

Gene therapies offer powerful new options for addressing previously fatal or untreatable genetic diseases. Our investigators are at the forefront of pre-clinical research, vector development and clinical trials of gene therapies for a broad range of pediatric disorders.

Gene therapy news
blast cells in leukemia medical science concept

Pediatric cancer

Childhood cancers are driven by different genetic changes than adult cancers. Our scientists interrogate their complex origins with advanced genomic and drug screening techniques, and partner with clinical researchers to drive a new generation of pediatric cancer trials.

Cancer research news
Hands held in the shape of a brain, covered by EEG leads


Understanding the nervous system at the genetic, molecular, cellular and systems level opens opportunities to tackle hard-to-treat disorders. From disease modeling in human neurons to clinical trials for genetic conditions, our basic and clinical scientists collaborate closely.

Neuroscience news


Next-generation DNA sequencing, chromosomal microarray, gene-expression studies, gene editing, genome-wide association studies and other tools are bringing fresh insights into the basic biology of disease, from common conditions like obesity to the exceedingly rare.

Genetics news
microscope image of b cell clones from the spleen


New tools are fueling a burst of discoveries in immune deficiency, allergy, autoimmunity and inflammatory disease. We are learning new immunization strategies and how the immune system influences diseases not classically seen as immunologic, from neurodegenerative disorders to heart disease.

Immunology news
Alpha-type retinal ganglion cells (RGCs) in part of an intact mouse retina.

Regenerative medicine

Our labs are creating pluripotent stem cells for disease modeling and drug screening, investigating natural regenerative processes with cellular “barcodes,” and developing cell-based therapies and testing chemical factors that stimulate regeneration pathways.

Regenerative medicine news

Boston Children's Hospital Innovation

BCH Innovation News @BCH_Innovation 3/18/2019 9:00:01 PM
Boston Children’s was included in a @Forbes roundup of powerful innovation in healthcare for the use of #3D models…
BCH Innovation News @BCH_Innovation 3/18/2019 6:29:32 PM
Boston Children’s Moritz Kraemer, PhD and an international team of researchers mapped how global warming could beco…
BCH Innovation News @BCH_Innovation 3/18/2019 6:29:31 PM
New studies establish a baseline for #health and #disease in newborns that may help measure responses to medical in…
BCH Innovation News @BCH_Innovation 3/18/2019 4:59:53 PM
.@xpress_medical reports on new research from Boston Children’s Martha Murray, MD, that reveals encouraging results…
BCH Innovation News @BCH_Innovation 3/18/2019 12:59:53 PM
To combat disease commonly carried by #mosquitoes, @Oxitec developed a method of #genetically modifying the pests s…
BCH Innovation News @BCH_Innovation 3/15/2019 8:59:52 PM
This new program at @Yale counsels parents living with an anxious child as an innovative way to treat #anxiety diso…
Boston Children's Hospital has been named the #1 children's hospital in the nation by U.S. News and World Report for the fifth year in a row! It's an honor that we could not have achieved without you. On behalf of every member of our Boston Children's team, thank you for inspiring us to be bolder, dream bigger, and make the impossible possible for our patients and families.

Sandra L. Fenwick, CEO

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