TY - JOUR AU - Antony, Justin S. AU - Latifi, Ngadhnjim AU - Haque, A. K. M. Ashiqul AU - Lamsfus-Calle, Andrés AU - Daniel-Moreno, Alberto AU - Graeter, Sebastian AU - Baskaran, Praveen AU - Weinmann, Petra AU - Mezger, Markus AU - Handgretinger, Rupert AU - Kormann, Michael S. D. PY - 2018 DA - 2018/11/14 TI - Gene correction of HBB mutations in CD34+ hematopoietic stem cells using Cas9 mRNA and ssODN donors JO - Molecular and Cellular Pediatrics SP - 9 VL - 5 IS - 1 AB - β-Thalassemia is an inherited hematological disorder caused by mutations in the human hemoglobin beta (HBB) gene that reduce or abrogate β-globin expression. Although lentiviral-mediated expression of β-globin and autologous transplantation is a promising therapeutic approach, the risk of insertional mutagenesis or low transgene expression is apparent. However, targeted gene correction of HBB mutations with programmable nucleases such as CRISPR/Cas9, TALENs, and ZFNs with non-viral repair templates ensures a higher safety profile and endogenous expression control. SN - 2194-7791 UR - https://doi.org/10.1186/s40348-018-0086-1 DO - 10.1186/s40348-018-0086-1 ID - Antony2018 ER -