Treating sickle cell disease and β–thalassemia

Researchers used CRISPR/Cas9 to genetically alter hematopoietic stem and progenitor cells to mimic a disorder called hereditary persistence of fetal hemoglobin (HPFH), and found that the deletion of a portion of the β–globin locus resulted in high γ-globin gene expression, a hallmark of HPFH, in the resulting red blood cells; patients who are doubly heterozygous for HPFH and β-thalassemia or sickle cell disease display milder disease manifestation, and the method, which mimics HPFH, may lead to a potential therapy for patients with β–thalassemia or sickle cell disease. - Read at PNAS.org

Article #16-12075: “Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia,” by Lin Ye et al.