Article
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 - 1 Feb 2022
Liu Yang, Dahl Maria, Debnath Shubhranshu, Rothe Michael, Smith Emma M, Grahn Tan Hooi Min, Warsi Sarah, Chen Jun, Flygare Johan, Schambach Axel, Karlsson Stefan
Abstract excerpt
Diamond-Blackfan anemia (DBA) is an inherited bone marrow failure disorder in which pure red blood cell aplasia is associated with physical malformations and a predisposition to cancer. Twentyfive percent of patients with DBA have mutations in a gene encoding ribosomal protein S19 (RPS19). Our previous proof-of-concept studies demonstrated that DBA phenotype could be successfully treated using lentiviral vectors...
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