Sickle Cell Disease: Getting to the Root of the Problem
we’re taking this opportunity to raise awareness about this devastating disease, elevate voices in the community to share their experience living with the disease and highlight potential treatments scientists at Vertex Pharmaceuticals have been working
on. Read on to learn more about the underlying cause of SCD and how scientists are currently investigating a potential gene-editing therapy to address this serious disease.
the production of hemoglobin that can carry oxygen properly.
with SCD often experience symptoms and complications including pain crises, strokes, anemia, jaundice, and symptoms of heart failure.
their disease. Vertex’s investigational approach uses CRISPR/Cas9 gene-editing technology to turn on the body’s natural machinery to increase the production of hemoglobin that can carry oxygen properly. They’re bringing together bestin- class research teams, technologies, and manufacturing capabilities in the hopes of bringing potentially transformative therapies to patients, like people with SCD and beta thalassemia, as quickly as possible.
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