Scientific minds are imagining. Sophisticated machines are harnessing genetic data. An invisible universe of microbes is revealing new insights into disease. Discoveries are changing children’s worlds. Every second of every day is filled with wonder at The Children’s Hospital of Philadelphia Research Institute.
An international team of gene experts has identified a mutation that causes aplastic anemia, a serious blood disorder in which the bone marrow fails to produce normal amounts of blood cells.
Hematologists have long sought to reactivate fetal hemoglobin as a treatment for children and adults with sickle cell disease (SCD). Researchers at CHOP have manipulated key biological events in adult blood cells to produce a form of hemoglobin normally absent after the newborn period.
An international team of researchers, including several investigators from The Children's Hospital of Philadelphia's Center for Applied Genomics, recently discovered a gene associated with a high risk of severe childhood asthma. The specific gene variant may be an actual cause of this form of asthma, a leading cause of hospitalization in young children.