Safety and Efficacy of CRISPR/Cas9 based Gene edited Hematopoietic Stem and Progenitor Cells in Hemoglobinopathies

Sickle cell disease is one of the most prevalent diseases worldwide. Even optimal conventional care has demonstrated inferiority compared to curative options such as hematopoietic stem cell transplantation. Gene editing promises an elegant alternative cure to all patients lacking a matched donor, potentially avoiding many of the transplantrelated complications. | Selim Corbacioglu is Professor for Pediatric and Adolescent Medicine as well as Director of the department for Pediatric Hematology, Oncology and Hematopoietic Stem-Cell Transplantation at the University Hospital Regensburg. 2019, he was able to succesfully treat a beta-thalassemia-patient by means of the CRISPR/Cas9 gene therapy for the first time.