Genetically modified induced pluripotent cells for treatment of sickle cell anemia

The aim of this study was to determine the therapeutic applicability of induced pluripotent stem (iPS) cells in treating sickle cell anemia. iPS cells derived from adult fibroblasts of a humanized knock-in mouse model of sickle cell anemia, were genetically modified to correct the sickle cell allele by homologous recombination, using electroporation. Then, the cells were differentiated into hematopoietic progenitor cells and transplanted into the sickle cell anemia mouse model to correct the sickle cell phenotype.