August 13, 2021

Sickle cell disease (SCD) is associated with complications that increase the risk of early death. In recent decades of SCD research, multiple interventions have proven successful in reducing premature mortality in infants and children with SCD. However, adults with SCD still have a high risk of early mortality, with an average life expectancy of 54...

Clinical manifestations of sickle cell disease, such as hemolytic anemia, pain, and progressive organ damage, can be alleviated by high levels of erythrocyte fetal hemoglobin (HbF). The BCL11A gene represses γ-globin expression and adult erythrocyte fetal hemoglobin production. This study, published in the New England Journal of Medicine, focuses on the genetic silencing of BCL11A, resulting...