Articles

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

Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, Sweden;
Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, Sweden;
Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, Sweden;
Institute of Experimental Hematology, Hannover Medical School, Germany;;LOEWE Research Group for Gene Modification in Stem Cells, Paul-Ehrlich-Institute, 63225 Langen, Germany
Institute of Experimental Hematology, Hannover Medical School, Germany;
Institute of Experimental Hematology, Hannover Medical School, Germany;
Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, Sweden;
Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, Sweden;
Vol. 99 No. 12 (2014): December, 2014 https://doi.org/10.3324/haematol.2014.111195