Liu Y, Dahl M, Debnath S, Rothe M, et al. 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. Haematologica 2021 Jan 14. doi: 10.3324/haematol.2020.269142.
PMID: 33440921