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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.

Learn more about mapping spinal-projecting neurons

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.

Learn more about sickle cell gene therapy

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.

Learn more about Naloxone on demand