 | Vol. 12.36 – 14 September, 2021 |
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| To identify genes with the most selective expression in human HSCs, researchers profiled population- and single-cell transcriptomes of un-expanded and ex vivo cultured cord blood-derived hematopoietic stem and progenitor cells as well as peripheral blood, adult bone marrow, and fetal liver. [Blood] |
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 | PUBLICATIONSRanked by the impact factor of the journal |
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| The authors investigated how mutations in either ribosomal protein large or ribosomal protein small subunit genes selectively affected erythroid progenitor development and clinical phenotypes in Diamond-Blackfan anemia, a rare ribosomopathy with limited therapeutic options. [Science Translational Medicine] |
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| Scientists performed extensive single-cell transcriptomic analyses to map fate choices and gene expression patterns during hematopoietic differentiation of human PSCs and showed that oxidative metabolism was dysregulated during in vitro directed differentiation. [Science Advances] |
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| Investigators demonstrated that reduced red blood cell (RBC) survival rather than altered erythropoiesis was driving the development of anemia. The tumor-induced inflammatory and metabolic remodeling affected RBC integrity and augmented splenic phagocyte activity promoting erythrophagocytosis. [Science Advances] |
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| RNF5 inhibition decreased acute myeloid leukemia cell growth in culture, in patient-derived xenograft samples and in vivo, and delayed development of MLL-AF9–driven leukemogenesis in mice, prolonging their survival. [Nature Communications] |
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| Using BRAF-mutated myeloma as a model for resistance to precision medicine, the authors investigated if BRAF-mutated cancer cells had the ability to ensure their survival by rapidly adapting to BRAF inhibitor treatment. [Clinical Cancer Research] |
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| Researchers investigated the impact of PRC1.1 insufficiency on myelofibrosis. Transcriptome and chromatin immunoprecipitation sequence analysis showed the de-repression of PRC1.1 target genes in Pcgf1-deficient JAK2V617F hematopoietic progenitors and revealed Hoxa cluster genes as direct targets. [Leukemia] |
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| Scientists demonstrated by clonal assays and single-cell transcriptomics that rare hemogenic endothelium (HE) with functional HSC potential in the early murine embryo were distinct from more abundant HE with multilineage hematopoietic potential that failed to generate HSCs. [Cell Reports] |
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| Using single-cell transcriptomics, investigators defined gene signatures that distinguished nascent HSCs from embryonic blood progenitors. Applying a lineage-tracing approach to selectively track HSC output in situ, they found significantly delayed lymphomyeloid contribution. [Cell Reports] |
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| The authors reported long-term follow-up of the Phase Ib study of venetoclax and rituximab in patients with relapsed chronic lymphocytic leukemia (CLL), including outcomes with continuous or limited-duration therapy. [Blood] |
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| Avelumab was tolerable and demonstrated antitumor activity in heavily pretreated patients with classical Hodgkin lymphoma (cHL), suggesting that PD-L1 blockade may be sufficient for therapeutic benefit in cHL. [Blood Advances] |
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| Researchers conducted a Phase II study to test the activity of bortezomib and rituximab in combination with a pediatric-inspired regimen during induction therapy in newly diagnosed adolescents and adults with CD20+, Philadelphia-negative precursor B-ALL; bone marrow measurable residual disease negativity at the end of induction was the primary end point. [Blood Advances] |
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| The authors summarize the status of hematopoietic stem and progenitor cell (HSPC) gene editing, focusing on efficiency, genomic integrity, and long-term engraftment ability related to available genetic editing platforms and HSPC delivery methods. [Molecular Therapy] |
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| Despite advances in understanding the clinical and biological aspects of myeloid leukemia in children with Down syndrome (DS), little is known about the mechanisms of relapse. Upon relapse, patients face a poor outcome, and there is no consensus on treatment. Future studies need to be focused on this challenging aspect of leukemia in children with DS. [Leukemia] |
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| Investigators discuss the role and importance of inflammasomes and immune pathways in myeloid malignancies, particularly myelodysplastic syndromes/acute myeloid leukemia, to better understand the disease pathophysiology and decipher the scope of therapeutic interventions. [Blood Cancer Journal] |
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| EdiGene, Inc. announced the first patient enrolled in multicenter Phase I clinical study of its investigational gene-editing hematopoietic stem cell therapy ET-01 for patients with transfusion dependent β-thalassemia at the Institute of Hematology and Blood Diseases Hospital. [EdiGene, Inc. (Business Wire, Inc.)] |
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| Jasper Therapeutics, Inc. announced the initiation of a Phase I/II clinical trial to evaluate JSP191, the company’s anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning agent prior to allogeneic transplant in patients with GATA2-related myelodysplastic syndromes. [Jasper Therapeutics, Inc. (Business Wire, Inc.)] |
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| Vor Biopharma announced that the US FDA has granted Fast Track designation to VOR33, the company’s lead engineered HSC therapeutic candidate for the treatment of acute myeloid leukemia (AML). [Vor Biopharma] |
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| Memorial Sloan Kettering Cancer Center has received a major grant from the US National Institutes of Health to conduct research related to leukemia. [Memorial Sloan Kettering Cancer Center] |
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| November 15 – 19, 2021 Virtual |
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| Houston Methodist Research Institute – Houston, Texas, United States |
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| University Hospital of Bern – Bern, Switzerland |
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| Banner MD Anderson Cancer Center – Gilbert, Arizona, United States |
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| Lund University – Lund, Sweden |
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| Sidney Kimmel Cancer Center at Jefferson Health – Philadelphia, Pennsylvania, United States |
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