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Medical Daily
Medical Daily
Elena Vega

The World's First CRISPR Gene Therapy Just Got Approved for Children as Young as 2 with Sickle Cell Disease

Why This Matters

Sickle cell disease begins doing damage from birth. The warped, crescent-shaped red blood cells it produces start blocking blood vessels in infancy and early childhood, causing painful crises, strokes, silent brain injuries, kidney damage, and progressive organ failure before a child is old enough to describe their symptoms. Most disease-modifying therapy has historically waited until patients were older, leaving the youngest children to absorb years of preventable damage before intervention became possible.

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