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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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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High Integrity and Fidelity of Long-Term Cryopreserved Umbilical Cord Blood for Transplantation

Researchers demonstrated the recovery of viable total nucleated cells and CD34+ cells, as well as the MNC viability in 20-year-old cryopreserved cord blood (CB) units in a CB bank.
[Journal of Clinical Medicine]
Kim, G.-H., Kwak, J., Kim, S. H., Kim, H. J., Hong, H. K., Jin, H. J., Choi, S. J., Oh, W., & Um, S. (2021). High Integrity and Fidelity of Long-Term Cryopreserved Umbilical Cord Blood for Transplantation. Journal of Clinical Medicine, 10(2), 293. https://doi.org/10.3390/jcm10020293 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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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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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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EdiGene Announces Approval of Its IND Application for Its CRISPR/Cas 9 Gene-Editing Hematopoietic Stem Cell Therapy ET-01 in β-thalassemia by China National Medical Products Administration

EdiGene, Inc. announced the Center for Drug Evaluation of China National Medical Products Administration has approved the company’s Investigational New Drug (IND) application for ET-01, an investigational CRISPR/Cas 9 gene-editing therapy for patients with transfusion dependent β-thalassemia.
[EdiGene, Inc.]
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Biologics License Application for Narsoplimab in HSCT-TMA Accepted for Priority Review by US FDA

Omeros Corporation announced that the Biologics License Application for narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy has been accepted for filing by the FDA.
[Omeros Corporation]
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Essential Role of Autophagy in Protecting Neonatal Hematopoietic Stem Cells from Oxidative Stress in a p62-Independent Manner

To investigate the effects of Atg5 deletion on stage-specific hematopoietic stem cell (HSC) functions, scientists compared the repopulating capacity of HSCs in Atg5f/f;Vavi-cre mice from postnatal day 0–7 weeks of age.
[Scientific Reports]
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EGR1 Is a Gatekeeper of Inflammatory Enhancers in Human Macrophages

Researchers analyzed the molecular determinants of monocytic and macrophagic commitment by profiling the EGR1 transcription factor. EGR1 is essential for monopoiesis and binds enhancers that regulate monocytic developmental genes such as CSF1R.
[Science Advances]
EGR1 is a gatekeeper of inflammatory enhancers in human macrophages | Science Advances. (n.d.). Retrieved January 18, 2021, from https://advances.sciencemag.org/content/7/3/eaaz8836 Cite
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Consensus Opinion on Immune-Mediated Cytopenias after Hematopoietic Cell Transplant for Inherited Metabolic Disorders

The authors summarize the literature, provide an approach to better understanding of the possible etiology of immune mediated cytopenias, and propose a diagnostic and management plan for patients with inherited metabolic disorders who develop single or multi-lineage cytopenias after hematopoietic stem cell transplantation.
[Bone Marrow Transplantation]
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