Gene therapy cures the anemia and lethal bone marrow failure in mouse model for RPS19-deficient Diamond-Blackfan anemia.

Pekka Jaako, Shubhranshu Debnath, Karin Olsson, Ute Modlich, Michael Rothe, Axel Schambach, Johan Flygare, Stefan Karlsson

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Biochemistry, Genetics and Molecular Biology

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