Project description:Bulk RNA-seq data of Lin-CD34+ hematopoietic stem and progenitor cells derived from bone marrow of healthy donors and untreated aplastic anemia patients
Project description:Transcriptome analysis of hematopoietic stem and progenitor cells (HSPCs) and T cells collected from bone marrow and peripheral blood of healthy donors and aplastic anemia patients untreated or response to immunosuppressive therapy.
Project description:CD3+ T cells derived from bone marrow aspirates and peripheral blood samples of newly diagnosed AA patients and healthy volunteers were analysed using Affymetrix HG_U133A GeneChips. Additionally, two patients were studied after achieving a partial remission (post-Therapy). Experiment Overall Design: Altogether, we analysed 8 different samples. Pool I contained three female and one male patients, 19 to 70 years old, suffering from (severe) aplastic anemia. From these four donors, samples derived from bone marrow aspirates and from peripheral blood, were pooled on total-RNA basis. Pool II consisted of additional two patients, one male (64 years old) and one female (70 years old). From both donors, total-RNA was extracted and pooled from CD3+ T cells derived either from bone marrow or peripheral blood, each prior and post therapy. Healthy control pools included three donors for the peripheral blood sample and two donors for bone marrow derived T cells.
Project description:Acquired aplastic anemia (AA) is an immune-mediated disease with active destruction of hematopoietic stem and progenitor cells by the cytotoxic T-cells in bone marrow. Aberrant expression of microRNAs in T-cells results in development of some autoimmune diseases.Screening the potential miRNAs which may play regulatory role in T cells of AA is meaningful to explore the mechanism of AA. We used microarrays to screen the differential expression pattern of miRNAs in T cells of aplatic anemia patients to find the potential regulatory miRNAs.
Project description:Fanconi anemia (FA) is a rare inherited disease complicated by aplastic anemia. There is evidence that hematopoietic stem cells have lost self replicative capacity and undergo apoptosis when exposed to inhibitory cytokines including interferon gamma and tumor necrosis factor-alpha. We used gene expression microarrays to identify transcriptomal differences between bone marrow cells from normal volunteers and from children and adults with Fanconi anemia Experiment Overall Design: Fanconi anemia patients were identified using mitomycin C and/or diepoxybutane chromosomal breakage analysis. Eleven normal volunteers and 21 FA patients were studied. All FA patients with cytogenetic evidence of clonal evolution were excluded. All FA patients with acute leukemia were excluded. RNA was prepared from freshly obtained low density mononuclear cell fractions.
Project description:Fanconi anemia (FA) is a rare inherited disease complicated by aplastic anemia. There is evidence that hematopoietic stem cells have lost self replicative capacity and undergo apoptosis when exposed to inhibitory cytokines including interferon gamma and tumor necrosis factor-alpha. We used gene expression microarrays to identify transcriptomal differences between bone marrow cells from normal volunteers and from children and adults with Fanconi anemia
Project description:<p>The most successful treatment for aplastic anemia is bone marrow transplantation. However, few patients are eligible for this procedure. For others, treatment usually consists of immunosuppressive agents, such as antithymocyte globulin (ATG) and cyclosporine. Unfortunately, even with immunosuppressive therapy, relapse is common. New combinations of medications may offer alternative and more effective treatment options. Sirolimus and cyclosporine are two drugs routinely used to suppress the immune system and prevent rejection in patients who have received organ transplants. While cyclosporine has been proven effective for treating aplastic anemia, sirolimus has not been tested for this disease. This study will evaluate the safety and efficacy of sirolimus in combination with cyclosporine for treating individuals with aplastic anemia that have not responded to other treatments.</p> <p>This study will last at least 6 months. Participants will first be screened to verify diagnosis of aplastic anemia. The screening will include a physical examination, blood test, bone marrow biopsy from the pelvic bone, and review of medications and medical history. Individuals who are eligible will then start the first treatment period. Participants will receive two medications: cyclosporine will be taken twice a day and sirolimus will be taken once a day. Depending on side effects, the dose of either drug may be temporarily stopped or lowered. On Day 1, blood will be drawn and females will undergo a pregnancy test. Subsequent study visits will occur weekly for the first month, every 2 weeks for 2 months, and then once a month for the remainder of the study. Each visit will include a physical examination, vital sign assessment, and review of side effects and medications. Blood tests will be performed weekly for the first 3 weeks, and then every 2 weeks.</p> <p>After 6 months of treatment, if a participant has shown improvements in disease status without major side effects, the treatment will continue. Over time the doses may be lowered. If a participant has not improved while on the study medication, treatment will stop at 6 months. Whenever treatment is discontinued, the participant will again undergo a physical examination, blood tests, and bone marrow biopsy.</p>
Project description:Gene expression analysis from erythroid progenitors (CD34+/CD71(high)/CD45- mononuclear cells from the bone marrow) of patients with Diamond-Blackfan anemia (due to RPS19 mutations) and control individuals.
Project description:The effect of gene therapy in the Hematopoietic stem cells of patients with fanconi anemia has been analyzed. For this purpose 3 fanconi patients bone marrow and a healthy one has been aspirated and CD34+ cells sorted. The data was processed using the usual 10X protocol
