Modeling, Optimization, and Comparable Efficacy of T Cell and Hematopoietic Stem Cell Gene Editing for Treating Hyper‐IgM Syndrome

Investigators developed a one‐size‐fits‐all editing strategy for effective T‐cell correction, selection, and depletion and investigated the therapeutic potential of T‐cell and hematopoietic stem/progenitor cell therapies in the hyper‐IgM syndrome mouse model.
[EMBO Molecular Medicine]
Vavassori, V., Mercuri, E., Marcovecchio, G. E., Castiello, M. C., Schiroli, G., Albano, L., Margulies, C., Buquicchio, F., Fontana, E., Beretta, S., Merelli, I., Cappelleri, A., Rancoita, P. M., Lougaris, V., Plebani, A., Kanariou, M., Lankester, A., Ferrua, F., Scanziani, E., … Genovese, P. (2021). Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome. EMBO Molecular Medicine, n/a(n/a), e13545. https://doi.org/10.15252/emmm.202013545 Cite
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Latest Culture Techniques: Cracking the Secrets of Bone Marrow to Mass-Produce Erythrocytes and Platelets Ex Vivo

Scientists discuss the latest culture techniques and technological approaches to obtain functional platelets and erythrocytes ex vivo.
[Haematologica]
Buduo, C. A. D., Aguilar, A., Soprano, P. M., Bocconi, A., Miguel, C. P., Mantica, G., & Balduini, A. (2020). Latest culture techniques: cracking the secrets of bone marrow to mass-produce erythrocytes and platelets <i>ex vivo. Haematologica. https://doi.org/10.3324/haematol.2020.262485 Cite
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Selective Antisense Oligonucleotide Inhibition of Human IRF4 Prevents Malignant Myeloma Regeneration via Cell Cycle Disruption

Using diverse pre-clinical models, the authors investigated the role of interferon regulatory factor 4 (IRF4) in myeloma progenitor regeneration.
[Cell Stem Cell]
Selective antisense oligonucleotide inhibition of human IRF4 prevents malignant myeloma regeneration via cell cycle disruption: Cell Stem Cell. (n.d.). Retrieved January 21, 2021, from https://www.cell.com/cell-stem-cell/fulltext/S1934-5909(20)30601-9 Cite
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SUMOylation Disassembles the Tetrameric Pyruvate Kinase M2 to Block Myeloid Differentiation of Leukemia Cells

Scientists found that SUMOylation of the M2 isoform of pyruvate kinase, a rate-limiting glycolytic enzyme catalyzing the dephosphorylation of phosphoenolpyruvate to pyruvate, is prevalent in a variety of leukemic cell lines as well as primary samples from patients with leukemia through multiple-reaction monitoring based targeted mass spectrometry analysis.
[Cell Death & Disease]
Xia, L., Jiang, Y., Zhang, X.-H., Wang, X.-R., Wei, R., Qin, K., & Lu, Y. (2021). SUMOylation disassembles the tetrameric pyruvate kinase M2 to block myeloid differentiation of leukemia cells. Cell Death & Disease, 12(1), 1–13. https://doi.org/10.1038/s41419-021-03400-9 Cite
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The TRACE-Seq Method Tracks Recombination Alleles and Identifies Clonal Reconstitution Dynamics of Gene Targeted Human Hematopoietic Stem Cells

The authors describe Tracking Recombination Alleles in Clonal Engraftment using sequencing (TRACE-Seq), a methodology that utilizes barcoded AAV6 donor template libraries, carrying in-frame silent mutations or semi-randomized nucleotides outside the coding region, to track the in vivo lineage contribution of gene-targeted hematopoietic stem and progenitor cell clones.
[Nature Communications]
Sharma, R., Dever, D. P., Lee, C. M., Azizi, A., Pan, Y., Camarena, J., Köhnke, T., Bao, G., Porteus, M. H., & Majeti, R. (2021). The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells. Nature Communications, 12(1), 472. https://doi.org/10.1038/s41467-020-20792-y Cite
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Interplay between Cofactors and Transcription Factors in Hematopoiesis and Hematological Malignancies

Investigators summarize the current knowledge regarding the models and functions of cofactor–transcription factor interplay in physiological hematopoiesis and highlight their implications in the etiology of hematological malignancies.
[Signal Transduction and Targeted Therapy]
Wang, Z., Wang, P., Li, Y., Peng, H., Zhu, Y., Mohandas, N., & Liu, J. (2021). Interplay between cofactors and transcription factors in hematopoiesis and hematological malignancies. Signal Transduction and Targeted Therapy, 6(1), 1–16. https://doi.org/10.1038/s41392-020-00422-1 Cite
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Selective Antisense Oligonucleotide Inhibition of Human IRF4 Prevents Malignant Myeloma Regeneration via Cell Cycle Disruption

Using diverse pre-clinical models, the authors investigated the role of interferon regulatory factor 4 (IRF4) in myeloma progenitor regeneration.
[Cell Stem Cell]
Mondala, P. K., Vora, A. A., Zhou, T., Lazzari, E., Ladel, L., Luo, X., Kim, Y., Costello, C., MacLeod, A. R., Jamieson, C. H. M., & Crews, L. A. (2021). Selective antisense oligonucleotide inhibition of human IRF4 prevents malignant myeloma regeneration via cell cycle disruption. Cell Stem Cell, 0(0). https://doi.org/10.1016/j.stem.2020.12.017 Cite
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Genome-Wide Analysis of Pseudogenes Reveals HBBP1’s Human-Specific Essentiality in Erythropoiesis and Implication in β-Thalassemia

Extensive functional assays demonstrated that HBBP1 was essential for erythropoiesis by binding the RNA-binding protein, HNRNPA1, to upregulate TAL1, a key regulator of erythropoiesis.
[Developmental Cell]
Ma, Y., Liu, S., Gao, J., Chen, C., Zhang, X., Yuan, H., Chen, Z., Yin, X., Sun, C., Mao, Y., Zhou, F., Shao, Y., Liu, Q., Xu, J., Cheng, L., Yu, D., Li, P., Yi, P., He, J., … Yu, J. (2021). Genome-wide analysis of pseudogenes reveals HBBP1’s human-specific essentiality in erythropoiesis and implication in β-thalassemia. Developmental Cell, 0(0). https://doi.org/10.1016/j.devcel.2020.12.019 Cite
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BMP-2 and VEGF-A modRNAs in Collagen Scaffold Synergistically Drive Bone Repair through Osteogenic and Angiogenic Pathways

Investigators probed the potential of bone marrow stem cells engineered with chemically modified mRNAs encoding the hBMP-2 and VEGF-A gene to therapeutically heal bone.
[Communications Biology]
Geng, Y., Duan, H., Xu, L., Witman, N., Yan, B., Yu, Z., Wang, H., Tan, Y., Lin, L., Li, D., Bai, S., Fritsche-Danielson, R., Yuan, J., Chien, K., Wei, M., & Fu, W. (2021). BMP-2 and VEGF-A modRNAs in collagen scaffold synergistically drive bone repair through osteogenic and angiogenic pathways. Communications Biology, 4(1), 1–14. https://doi.org/10.1038/s42003-020-01606-9 Cite
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Hematopoietic Cell Transplant in Pediatric Acute Myeloid Leukemia after Similar Upfront Therapy; A Comparison of Conditioning Regimens

Researchers retrospectively analyzed the impact of Busulfan–Cyclophosphamide, Busulfan–Cyclophosphamide–Melphalan and Clofarabine–Fludarabine–Busulfan in pediatric AML-patients, with similar upfront leukemia treatment, receiving an hematopoietic cell transplant between 2010 and 2015.
[Bone Marrow Transplantation]
Versluys, A. B., Boelens, J. J., Pronk, C., Lankester, A., Bordon, V., Buchner, J., Ifversen, M., Jackmann, N., Sundin, M., Vettenranta, K., Abrahamson, J., & Mellgren, K. (2021). Hematopoietic cell transplant in pediatric acute myeloid leukemia after similar upfront therapy; a comparison of conditioning regimens. Bone Marrow Transplantation, 1–7. https://doi.org/10.1038/s41409-020-01201-w Cite
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Vor Announces FDA Clearance of IND Application for VOR33

Vor Biopharma announced that the FDA has cleared the company’s Investigational New Drug (IND) application for VOR33, an engineered hematopoietic stem cell therapy candidate being developed for the treatment of acute myeloid leukemia.
[Vor Biopharma]
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