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.
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.
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.
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.
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.
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.
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.
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.
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