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.

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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 1/20/2019 5:59:01 PM
Wow! New technique #images the #brain like never before—in days rather than decades:
BCH Innovation News @BCH_Innovation 1/20/2019 5:26:55 PM
The #microbiome works as a #biologicalclock: Gut bacteria were able to predict a person’s age to within 4 years:…
BCH Innovation News @BCH_Innovation 1/18/2019 10:00:01 PM
A long-term study of #institutionalized children in Romania reveals impaired memory and executive functioning at ag…
BCH Innovation News @BCH_Innovation 1/18/2019 4:29:32 PM
Boston Children’s & @MedumoInc, a startup with a #patientnavigation platform, joined forces to offer a pediatric ca…
BCH Innovation News @BCH_Innovation 1/17/2019 9:59:55 PM
Boston Children’s Larry Benowitz, PhD, offers insight on an external study that discovered a method to bridge damag…
BCH Innovation News @BCH_Innovation 1/17/2019 8:59:31 PM
.@Science_Daily reports newly identified molecule linked to #dilatedcardiomyopathy helps doctors develop specific t…
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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