Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector

Yang Liu, Maria Dahl, Shubhranshu Debnath, Michael Rothe, Emma Smith, Hooi Min Tan Grahn, Sarah Warsi, Jun Chen, Johan Flygare, Axel Schambach , Stefan Karlsson

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Medicine & Life Sciences