| Vol. 14.11 – 21 March, 2023 |
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| Researchers described the results of the first-in-human Phase I clinical trial investigating revumenib, a potent and selective oral inhibitor of the menin–KMT2A interaction, in patients with relapsed or refractory acute leukemia. [Nature] |
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| PUBLICATIONSRanked by the impact factor of the journal |
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| Using single cell transcriptomics, scientists longitudinally profiled bone marrow and blood samples from patients with aplastic anemia undergoing allogeneic HSC transplantation with G-CSF–mobilized grafts. [Science Immunology] |
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| Investigators showed that in contrast to most cells that primarily utilize the proteasome to degrade misfolded proteins, HSCs preferentially traffic misfolded proteins to aggresomes in a Bag3-dependent manner and depend on aggrephagy, a selective form of autophagy, to maintain proteostasis in vivo. [Cell Stem Cell] |
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| Researchers revealed a Plek2/Akt complex that drove cell proliferation and could be targeted by a class of antiproliferative compounds for myeloproliferative neoplasm therapy. [Journal Of Clinical Investigation] |
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| Scientists found a conserved epitranscriptomic program that steers SF3B1 levels to counteract leukemogenesis. Their analysis of human and murine pre-leukemic myelodysplastic syndrome (MDS) cells revealed regulation of SF3B1 protein abundance, which affected MDS-to-leukemia progression in vivo. [Molecular Cell] |
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| Through gene expression profiling, investigators found HES6 as an abundant cofactor expressed at gene level during human erythropoiesis. [Nucleic Acids Research] |
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| Scientists used an in vitro differentiation system and RNA-sequencing to compare monocyte-derived dendritic cells from Langerhans cell histiocytosis patients to those derived from healthy controls or patients with Crohn’s disease, a non-histiocytic inflammatory disease. [Haematologica] |
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| Researchers studied the switch/sugar non-fermenting subunit BCL7A, which was found to be recurrently mutated in lymphomas, but whose role in acute myeloid malignancies is currently unknown. [Biomarker Research] |
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| Clinical effect of donor-derived natural killer cell infusion after HLA-haploidentical hematopoietic cell transplantation (HCT) was evaluated in high-risk myeloid malignancy in Phase II, randomized trial. [Leukemia] |
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| The authors review the observational and mechanistic studies describing the connection between clonal hematopoiesis (CH) and pathological immune dysfunction, as well as the effects of CH-associated genetic alterations on the function of myeloid and lymphoid cells. [Nature Reviews Immunology] |
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| Disc Medicine, Inc. announced a collaboration with the National Heart Lung and Blood Institute of the National Institutes of Health to evaluate bitopertin, a therapeutic candidate designed to modulate heme biosynthesis, in a Phase II clinical study of patients with DBA. [Disc Medicine, Inc.] |
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| April 23 – 26, 2023 Paris, France |
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| University of Florida – Gainesville, Florida, United States |
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| Columbia University – New York City, New York, United States |
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| King’s College London – Strand, England, United Kingdom |
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| Lund University – Lund, Sweden |
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| Dana-Farber Cancer Institute – Boston, Massachusetts, United States |
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