Fetal vs Adult Hemoglobin: The Divide at the Heart of Gene Therapy

For decades, sickle cell disease has stood as one of medicine’s starkest unmet needs: one genetic mutation leading to painful crises, shortened lives, and limited treatment options. Patients with transfusion-dependent thalassemia faced a similar fate, relying on lifelong transfusions and iron chelation with no durable cure in sight. Bone marrow transplant was a hope for a few with compatible donors, but for the rest, treatment consisted of symptom management…