title: Diamond blackfan anemia stem cells fail to repopulate erythropoiesis in NOD/SCID mice.
abstract: Diamond Blackfan Anemia (DBA) is a congenital disorder characterized by decreased red blood cell production and developmental abnormalities. We herein show that DBA progenitors produced lower numbers of phenotypically normal erythroid colonies in vitro, whereas nonerythroid colonies were normally abundant and developed. To determine whether DBA stem cells are capable of producing early erythroid, monocyto-granulocytic, and lymphoid progenitors in vivo we used a mouse xenotransplantation model. We demonstrate that DBA stem cells poorly repopulated erythroid progeny in NOD/SCID mice, whereas the monocyto-granulocytic and lymphoid progenies were repopulated normally. Therefore, we conclude that disordered DBA erythropoiesis may be a result of defective erythroid-lineage commitment and maintenance of early erythroid progenitors.
authors.affiliation: Department of Pathophysiology, Charles University, First Medical Faculty, Prague, Czech Republic. zivny@cber.FDA.gov
authors.forename: Jan
authors.initials: J
authors.lastname: Zivny
authors.forename: Jaroslav
authors.initials: J
authors.lastname: Jelinek
authors.forename: Dagmar
authors.initials: D
authors.lastname: Pospisilova
authors.forename: Magdalena
authors.initials: M
authors.lastname: Plasilova
authors.forename: Emanuel
authors.initials: E
authors.lastname: Necas
authors.forename: Tomas
authors.initials: T
authors.lastname: Stopka
mesh_terms: D029503:Anemia, Diamond-Blackfan / Q000209:etiology / Q000473:pathology*; D000818:Animals; D018952:Antigens, CD34; D016022:Case-Control Studies; D002648:Child; D003114:Colony-Forming Units Assay; D004920:Erythropoiesis; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000473:pathology*; D006801:Humans; D019891:Leukopoiesis; D051379:Mice; D016688:Mice, Inbred NOD; D023421:Models, Animal; D014183:Transplantation, Heterologous
chemical_list: D018952:Antigens, CD34