Research

Research

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.

Hearts get a boost from mitochondrial transplantation

For decades, cardiac researcher James McCully, PhD, has been spellbound by the idea of using mitochondria as a therapy to boost heart function in patients who’ve experienced ischemia as a result of heart defects, coronary artery disease or heart attack. Finally, a clinical trial is bringing his vision — a therapy called mitochondrial transplantation — to life. McCully and heart surgeon Sitaram Emani, MD, are giving the therapy to the sickest of the sick patients in Boston Children’s cardiac ICU and seeing transformative results.

Avery-mitochondria

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

Neuroscience

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

Genetics/Genomics

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

Immunology

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 9/24/2018 8:59:50 PM
What is an #innovation dashboard? For Boston Children's, it is a tool used to track capacity in real-time and maxim… https://t.co/R77eAfz0i5
BCH Innovation News @BCH_Innovation 9/24/2018 8:59:50 PM
How clinicians interact with the #EHR is being upended by #voice tech. Experience live demos of the disruptors, inc… https://t.co/hFCYmrC5o5
BCH Innovation News @BCH_Innovation 9/24/2018 4:59:35 PM
Join us this fall for the VoiceDotHealth Summit: a full-day, pre-conference event of Connected Health Conference. C… https://t.co/rjM3oBk31H
BCH Innovation News @BCH_Innovation 9/24/2018 4:00:01 PM
“We’ve very seldom seen a drug as active as retinoic acid,” explains Jeff Kaufman of the Adenoid Cystic Carcinoma R… https://t.co/jm8l8pS08Z
BCH Innovation News @BCH_Innovation 9/24/2018 2:59:44 PM
Our immune cells could hold teach us how to modulate the body’s immune response to infection:… https://t.co/QIR2WCpW73
BCH Innovation News @BCH_Innovation 9/24/2018 2:40:17 PM
Antibiotic-resistant bacteria, infecting >100 people, have been linked to pet store puppies given #antibiotics prop… https://t.co/Zi527PjF3i
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, President and CEO

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