[HTML][HTML] Bone marrow transplantation without myeloablative conditioning in a mouse model for Diamond-Blackfan anemia corrects the disease phenotype
M Dahl, S Warsi, Y Liu, S Debnath, M Billing… - Experimental …, 2021 - Elsevier
Highlights•Reduced-intensity conditioning before hematopoietic stem cell transplantation.•
Anemia and bone marrow failure corrected in Rps19-deficient mice after
transplant.•Syngeneic wild-type cells have a selective advantage in Rps19-deficient
marrows.•Progressive increase in donor-derived cells was observed only in Rps19-deficient
recipients.Diamond-Blackfan anemia (DBA) is a congenital erythroid hypoplasia caused by
a functional haploinsufficiency of genes coding for ribosomal proteins. Among these genes …
Anemia and bone marrow failure corrected in Rps19-deficient mice after
transplant.•Syngeneic wild-type cells have a selective advantage in Rps19-deficient
marrows.•Progressive increase in donor-derived cells was observed only in Rps19-deficient
recipients.Diamond-Blackfan anemia (DBA) is a congenital erythroid hypoplasia caused by
a functional haploinsufficiency of genes coding for ribosomal proteins. Among these genes …
