 | | Vol. 12.38 – 28 September, 2021 |
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| Researchers applied an integrated genomic approach to a murine allelic series that modeled the two most common mutations in acute myeloid leukemia (AML): Flt3-ITD and Npm1c, then deconvoluted the contribution of each mutation to alterations of the epigenetic landscape and genome organization.
[Nature Genetics] |
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 | | PUBLICATIONSRanked by the impact factor of the journal |
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| Scientists demonstrated that osteoblastic cells in the bone marrow niche elaborated extracellular vesicles (EV) that were taken up by hematopoietic progenitor cells in vivo, and that upregulating EV transfer improved hematopoietic recovery from genotoxic injury and survival from fungal sepsis.
[Cell Stem Cell] |
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| The authors reported that post-transcriptional repression of the transcription factor ARID3A by miR-125b was a key event in megakaryoblastic leukemia pathogenesis, and showed that chromosome 21-encoded miR-125b synergized with Gata1s to drive leukemogenesis in this context.
[Blood] |
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| Disruption of the super-enhancer-associated gene transcription by THZ1, a covalent cyclin-dependent kinase 7 inhibitor, efficiently eradicated leukemia stem cells in retroviral BCR-ABL–driven chronic myelogenous leukemia mice while sparing normal hematopoietic stem cells.
[Science Translational Medicine] |
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| By combining Ribo-seq, mass spectrometry, and RNA-seq datasets, investigators discovered an oncomicropeptide, APPLE (a peptide located in ER), encoded by a non-coding RNA transcript in acute myeloid leukemia.
[Molecular Cell] |
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| Researchers generated zebrafish models deficient in Ufm1 and Ufl1 that exhibited telomere shortening associated with developmental delay, impaired hematopoiesis and premature aging.
[Science Advances] |
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| Since Wiskott–Aldrich Syndrome protein (WASp) is exclusively expressed in hematopoietic cells, scientists performed in silico screening to identify small molecule compounds that bound WASp and promoted its degradation.
[Nature Communications] |
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| Investigators showed that hematopoiesis and RNA splicing in U2af1 heterozygous knock-out mice was similar to control mice, but that deletion of the wild-type allele in U2AF1 heterozygous mutant expressing hematopoietic cells was lethal.
[Journal of Clinical Investigation] |
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| Human malignant cells distributed in the bioreactor system mimicking the spatial distribution found in native bone marrow tissue, where most hematopoietic stem and progenitor cells remained linked to the niches and mature cells were released to the circulation.
[Proceedings of the National Academy of Sciences of the United States of America] |
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| Spred1 knockout, regardless if occurred in HSCs or in endothelial cells, increased miR-126 in Lin−Sca-1+c-Kit+, a population enriched in leukemic stem cells (LSCs), resulting in expansion of LSCs, likely through hyperactivation of the MAPK/ERK pathway that augmented Bcl-2 expression and stability.
[Leukemia] |
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| A Phase I/II study evaluated the safety, tolerability, and anti-leukemic activity of uproleselan with mitoxantrone, etoposide, and cytarabine among patients with relapsed/refractory acute myeloid leukemia.
[Blood] |
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| Researchers aimed to modify the immune response in the long term after allogeneic bone marrow transplantation by using the proteasome inhibitor ixazomib at the late stages of the post-transplant period.
[Bone Marrow Transplantation] |
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| Scientists reported results of a prospective pilot trial evaluating the safety/feasibility of peri-transplant administration of ruxolitinib for myelofibrosis treatment. Peri-HCT ruxolitinib was safe and well-tolerated with the maximum tolerated dose determined as 10 mg BID, associated with dose-dependent PK and cytokine profile.
[Blood Advances] |
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| Investigators present a detailed overview of the most recent studies that present experimental evidence on how leukemic cells can metabolically rewire, more specifically the importance of OXPHOS in leukemic stem cells and treatment-resistant cells, and the current drugs available to target this process.
[Leukemia] |
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| The authors revisit biological characteristics and functions of infiltrating myeloid cells and discuss the recent advances in immunotherapies targeting infiltrating myeloid cells in glioblastoma .
[Oncogene] |
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| Scientists discuss the significant role of long non-coding RNAs in the proliferation, survival, differentiation, leukemic stem cells in acute myeloid leukemia and their involvement in different molecular pathways
[Journal of Cellular Physiology] |
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| JW Therapeutics announced that it has received the Investigational New Drug clearance from the National Medical Products Administration of China for a clinical trial of BCMA-targeted Chimeric Antigen Receptor T cell JWCAR129 in treating relapsed or refractory multiple myeloma.
[JW Therapeutics] |
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| AB Science SA announced that its clinical trial with AB8939 in adult patients with relapsed/refractory acute myeloid leukemia (AML) has been approved by Health Canada. The company will proceed with a Phase I/II study in patients with refractory and relapsed AML and refractory myelodysplastic syndrome.
[AB Science SA (Globe Newswire, Inc.)] |
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| bluebird bio, Inc. announced it has completed the rolling submission of its BLA to the US FDA for beti-cel gene therapy in adult, adolescent and pediatric patients with β-thalassemia who require regular red blood cell transfusions, across all genotypes.
[bluebird bio, Inc.] |
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| September 30 – October 1, 2021
Virtual |
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| | The University of New Mexico – Albuquerque, New Mexico, United States |
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| | Moores Cancer Center, UC San Diego – San Diego, California, United States |
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| | NHS Blood and Transplant – Oxford, England, United Kingdom |
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| | Cellectis – Paris, France |
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| | Houston Methodist Research Institute – Houston, Texas, United States |
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