CRISPR-Cas9 Gene Editing in Hematopoietic Stem Cells for Sickle Cell Disease Treatment

Sickle cell disease is caused by a single point mutation in the beta-globin gene. We developed a CRISPR-Cas9-based approach to correct this mutation in patient-derived hematopoietic stem cells. Our method achieved 67% correction efficiency with minimal off-target effects. Edited cells showed normal hemoglobin production and resistance to sickling under hypoxic conditions. These findings demonstrate the therapeutic potential of gene editing for inherited blood disorders.
