Forbes December 1, 2025
A new chapter is unfolding for patients with sickle cell disease and beta-thalassemia: breakthroughs in gene regulation are transforming how these conditions are treated. Emerging approaches focus on turning fetal hemoglobin back on by blocking the molecular “off switch” that normally silences it after birth. These directly target the regulatory pathways controlling hemoglobin expression. With the potential to reduce painful crises, organ damage and transfusion dependence, precision-driven gene and epigenetic therapies have the potential to offer a more targeted path for the millions affected worldwide.
Think of it like a nursery light switch. When a baby is born and starts to grow, it’s as if the sun comes up. Once this happens, you don’t need the overhead light anymore, and...
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