Orkin's research on the hemoglobin gene paved the way for a gene-based therapy. CRISPR technology enabled reactivating fetal hemoglobin, offering a life-changing cure for sickle cell patients.
The therapy involves editing diseased blood cells in a lab to produce fetal hemoglobin, eliminating the need for painful episodes and frequent blood transfusions associated with sickle cell disease.
Orkin aims to make the therapy more accessible by exploring methods to trigger fetal hemoglobin production using a pill, highlighting the ongoing commitment to improving treatment options.
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