About Us

Boston Children’s Hospital invests heavily in research because science saves lives. We are focused on accelerating new treatments for devastating diseases — our discoveries translate from the laboratory bench to the patient’s bedside, where they have been improving the health of children and adults since 1869. 

Today, our research enterprise is the world's largest and most highly-funded at a pediatric hospital, investing $355 million in expenditures in fiscal year 2017 alone. In FY2018, Boston Children’s was #1 in National Institutes of Health funding for all U.S. children’s hospitals, and #6 in funding for research institutes at any independent hospital. More than 3,000 basic, clinical and translational research employees work in our dedicated facilities, which total nearly 1 million square feet of space. 

Members of our research community include nine members of the National Academy of Sciences, 18 members of the National Academy of Medicine, 16 Howard Hughes Medical Institute Investigators and alumni, and eight Lasker Award recipients. In total, our research community publishes more peer-reviewed research in top scientific journals than the next 20 children’s hospitals combined — more than 3,000 annually since 2015.

Research discovery platforms

  • genetics and genomics
  • bioinformatics
  • large, diverse rare disease populations
  • biobank of tissues from patients with diverse disorders
  • multiple animal models
  • engineered tissues, including organs on chips
  • patient-derived, disease-specific cell lines
  • automated, high-throughput image analysis
  • proteomics, metabolomics, methylomics and other “omics”
  • gene editing with CRISPR/Cas9 technology
  • biomaterials and bioengineering
  • zebrafish drug discovery platforms
  • 3D human tissue construct for vaccine testing

Research discovery highlights

Recently, our scientists have:

  • restored hearing in deaf mice with gene therapy
  • showed how immune cells help shape brain connections during development, opening new angles on schizophrenia and Alzheimer’s disease
  • created blood stem cells in the lab
  • developed new drug delivery methods, like nanoparticles activated by light or ultrasound, drug eluting contact lenses and bioengineered blood vessels
  • used fetal cells from amniotic fluid to repair congenital defects in animal models
  • developed a one-time topical antibiotic treatment for otitis media
  • used biodegradable silk scaffolds to regenerate hollow structures like the bladder and esophagus.

Precision medicine initiatives

“Precision medicine” at Boston Children’s begins with discoveries at the lab bench and ends with customized care based a patient’s genetic profile and disease biology. That can mean an epilepsy drug targeted to a child’s specific mutation, a vaccine designed specifically for the newborn immune system or genetic sequencing to match cancer patients with the right therapies. A few of our initiatives:

  • The Manton Center for Rare Disease Research to help solve patients’ genetic mysteries and begin to develop treatments. We are also a member of the NIH’s Undiagnosed Diseases Network.
  • Gene therapy for sickle cell, severe immunodeficiency (SCID-X), adrenoleukodystrophy, relapsed acute lymphoblastic leukemia, Wiskott-Aldrich syndrome and a growing list of other conditions. Our scientists have been instrumental in developing safer delivery vectors.
  • BabySeq: Together with Brigham & Women’s Hospital, we are studying the impact of sequencing newborns’ DNA.
  • CLARITY Challenges: We sponsored two international crowdsourcing challenges to analyze and interpret genomic information to benefit patients.
  • Pharmacogenomics, reducing adverse drug reactions and improving children’s responses to therapy through genetic profiling
  • The Precision Link Biobank for Health Discovery, a library of blood, tissue and cells donated by thousands of patients and their families at Boston Children's Hospital to advance knowledge of health and disease.
  • Exome sequencing initiatives for epilepsy, inflammatory bowel disease and more.