Stefano Rivella, PhD, is developing a novel gene therapy that could help patients living with sickle cell disease and other debilitating red blood disorders.
This week’s stories span discovery (of a potential treatment for rare blood diseases), inspiration (from a mother who transformed grief over her daughter’s terminal disease into a foundation helping other families), and finding practical clinical insight (to distinguish a serious knee condition from a drug-treatable infection with similar symptoms).
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.
After 30 years of research, one surgeon is close to a breakthrough for sickle cell disease. It’s a good rule of thumb that when it comes to sick kids, the sooner a problem is identified and treated, the better the prognosis. Many of CHOP’s patients are toddlers, infants or even newborns. But one group of surgeons and researchers at CHOP works on a whole different timeline: They treat babies before they are even born.
