GIỚI THIỆU VỀ TẬP NÀY
Sickle cell disease is caused by a single mutation in the beta-globin gene that leads to painful crises, anemia, and organ damage. Despite advances in treatment, it remains a devastating and often overlooked global health challenge, particularly in low-resource settings where children frequently go undiagnosed until life-threatening complications arise. The PERICLES project is an ambitious research initiative exploring prenatal gene editing to correct sickle cell disease before birth by targeting fetal blood-forming stem cells. Panicos Shangaris, a clinical senior lecturer and consultant in maternal and fetal medicine at King’s College London, discusses what daily life looks like for people living with sickle cell disease today, the limits of existing therapies, and why treating the condition in utero could offer families a one-time, potentially curative therapy to prevent the disease before symptoms appear.
