Research at Boston Children's Hospital
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 biology coupled with world-class discovery platforms, including genetics and genomics, gene editing, bioinformatics, proteomics, bioengineering, image analysis, biobanks, disease-specific stem cell lines, and a range of animal models. We have special expertise in rare disease discovery, a robust Translational Research Program and large, diverse patient populations for clinical research and trials.
New leads for spinal cord injury: Mapping spinal-projecting neurons in the brain
Our scientists have pulled off a monumental task: Mapping and cataloguing all the neurons in the brain that connect to the spinal cord. The work may inform efforts to treat spinal cord injury.
Sickle cell gene therapy and boosting fetal hemoglobin: A 75-year history
The approval of the first gene editing therapy for sickle cell disease has a long backstory. Much science – starting 75 years ago – went into making creating this potentially curative treatment.
Naloxone on demand: Shining a light to reverse opioid overdose
This innovative technology could counter opioid overdoses when Narcan is unavailable. It involves advance, subcutaneous injections of light-activate nanoparticles containing naloxone.
Rising stars in research
Meet some of our scientists and the projects they hope to advance.