IPSC modeling of severe aplastic anemia reveals impaired differentiation and telomere shortening in blood progenitors.

ABSTRACT: Aplastic Anemia (AA) is a bone marrow failure (BMF) disorder, resulting in bone marrow hypocellularity and peripheral pancytopenia. Severe aplastic anemia (SAA) is a subset of AA defined by a more severe phenotype. Although the immunological nature of SAA pathogenesis is widely accepted, there is an increasing recognition of the role of dysfunctional hematopoietic stem cells in the disease phenotype. While pediatric SAA can be attributable to genetic causes, evidence is evolving on previously unrecognized genetic etiologies in a proportion of adults with SAA. Thus, there is an urgent need to better understand the pathophysiology of SAA, which will help to inform the course of disease progression and treatment options. We have derived induced pluripotent stem cell (iPSC) from three unaffect

SUBMITTER: Melguizo-Sanchis D 

PROVIDER: S-EPMC5833558 | biostudies-literature | 2018 Jan

REPOSITORIES: biostudies-literature