ObjectivesSevere aplastic anemia is characterized by a hypocellular bone marrow and peripheral cytopenia. Mesenchymal stem cells (MSCs) play a crucial role in haematopoietic stem cells (HSCs) development and the microenvironment suitable for haematopoiesis. Investigation of the therapeutic targets by paediatric patient-specific gene expression analysis of the MSCs can be important for diagnosis.MethodsThe study was based on freely available miRNA and host gene expression in NCBI GEO dataset.

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... roarray based gene expression profiles (GSE33812) of MSCs for five paediatric aplastic anaemia patients and healthy controls were generated using Agilent-014850 platform and the data was downloaded from the database.ResultsMSCs gene expression profiling distinguished between healthy controls, children with aplastic anemia. Angioteninogen (AGT) gene involved in ERK1/ERK2 cascade, cyotokine secretion, metabolic processes was strongly down-regulated among all the patients with aplastic anemia. Emerging role of various transcription factors binding to this gene was identified as a new avenue of therapeutic application.ConclusionsAs a potential diagnostic tool, patient-specific gene expression profiling of MSCs made it possible to make the difficult diagnosis of most patients with aplastic anemia.