Stanford Medicine scientists are launching a clinical trial of prenatal transplants, using stem cells from the mother, to treat a rare genetic disease called Fanconi anemia before a baby is born.
Stanford Medicine pediatric hematologist Agnieszka Czechowicz, MD, PhD, has devoted her research career to improving treatments for rare blood disorders. She’s an expert in Fanconi anemia, a genetic disease that interferes with DNA repair and blood cell production. By age 12, most people with the disease experience a life-threatening complication called bone marrow failure, in which the body’s blood-cell factory stops functioning.
