CRISPR/Cas9 Genome-Edited Autologous Hematopoietic Stem Cell Transplantation Therapies for Sickle Cell Disease

Advances in the application of CRISPR/Cas9 system in the study of hematologic diseases and cancer

“…HbS at high concentration are prone to form polymers so that leading to transformation of red blood cells (RBCs), and presenting sickle-rigidity or crescent shaped [42][43][44]. SCD can result in vascular occlusion, very severe multisystem organ dysfunction, and eventually threatening life expectancy of patients [45]. The only method for curing SCD is allogeneic hematopoietic stem cell transposition (HSCT).…”