Disease modelling of bone marrow failure syndromes using iPS cell-derived hematopoietic stem progenitor cells

Aplastic anemia (AA) is a life-threatening bone marrow failure (BMF) disorder, resulting in bone marrow hypoplasia, infection and hemorrhage, and severe peripheral pancytopenia. Although the most cases of AA are acquired and are associated with the autoimmune destruction of hematopoietic stem progenitor cells (HSPCs) in the BM, in some cases the BMF is caused by genetic or inherited anomalies that impair hematopoiesis [1]. The destruction or dysfunction of HSPCs in the BM of patients with BMFSs limits to study of these disorders since the use of conventional  in vitro HSPC culture or in vivo animal models for creating patient-specific disease modelling are technically impossible due to the unavailability of patient-derived HSPCs.

Source: Experimental Hematology - January 18, 2019 Category: Hematology Authors: Mahmoud I. Elbadry, J. Luis Espinoza, Shinji Nakao Source Type: research