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]
Nomura, N., Ito, C., Ooshio, T., Tadokoro, Y., Kohno, S., Ueno, M., Kobayashi, M., Kasahara, A., Takase, Y., Kurayoshi, K., Si, S., Takahashi, C., Komatsu, M., Yanagawa, T., & Hirao, A. (2021). Essential role of autophagy in protecting neonatal haematopoietic stem cells from oxidative stress in a p62-independent manner. Scientific Reports, 11(1), 1666. https://doi.org/10.1038/s41598-021-81076-z Cite
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Adipose Tissue Inflammation and Systemic Insulin Resistance in Mice with Diet-Induced Obesity Is Possibly Associated with Disruption of PFKFB3 in Hematopoietic Cells

Researchers examined a role for 6-phosphofructo-2-kinase/fructose-2,6-bisphosphatase 3 (PFKFB3) in hematopoietic cells in regulating white adipose tissue inflammation and systemic insulin sensitivity.
[Laboratory Investigation]
Zhu, B., Guo, X., Xu, H., Jiang, B., Li, H., Wang, Y., Yin, Q., Zhou, T., Cai, J. J., Glaser, S., Meng, F., Francis, H., Alpini, G., & Wu, C. (2021). Adipose tissue inflammation and systemic insulin resistance in mice with diet-induced obesity is possibly associated with disruption of PFKFB3 in hematopoietic cells. Laboratory Investigation, 1–13. https://doi.org/10.1038/s41374-020-00523-z Cite
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Adrenomedullin-CALCRL Axis Controls Relapse-Initiating Drug Tolerant Acute Myeloid Leukemia Cells

Calcitonin receptor-like receptor (CALCRL) knockdown impaired leukemic growth, decreased leukemic stem cell frequency, and sensitized to cytarabine in patient-derived xenograft models.
[Nature Communications]
Larrue, C., Guiraud, N., Mouchel, P.-L., Dubois, M., Farge, T., Gotanègre, M., Bosc, C., Saland, E., Nicolau-Travers, M.-L., Sabatier, M., Serhan, N., Sahal, A., Boet, E., Mouche, S., Heydt, Q., Aroua, N., Stuani, L., Kaoma, T., Angenendt, L., … Sarry, J.-E. (2021). Adrenomedullin-CALCRL axis controls relapse-initiating drug tolerant acute myeloid leukemia cells. Nature Communications, 12(1), 422. https://doi.org/10.1038/s41467-020-20717-9 Cite
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CD14+ Monocytes Repress Gamma Globin Expression at Early Stages of Erythropoiesis

Researchers showed cultured erythroblasts were a mix of HbA restrictive and HbA/HbF expressing cells and that the proportion of cells in the latter population depends on the starting material.
[Scientific Reports]
Heshusius, S., Heideveld, E., von Lindern, M., & van den Akker, E. (2021). CD14+ monocytes repress gamma globin expression at early stages of erythropoiesis. Scientific Reports, 11(1), 1507. https://doi.org/10.1038/s41598-021-81060-7 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]
Gupta, A. O., Jan Boelens, J., Ebens, C. L., Kurtzberg, J., Lund, T. C., Smith, A. R., Wagner, J. E., Wynn, R., Blazar, B. R., & Orchard, P. J. (2021). Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders. Bone Marrow Transplantation, 1–10. https://doi.org/10.1038/s41409-020-01179-5 Cite
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Co-Transcriptional Splicing Regulates 3′ End Cleavage during Mammalian Erythropoiesis

To determine how co-transcriptional splicing is integrated with transcription elongation and 3′ end formation in mammalian cells, investigators performed long-read sequencing of individual nascent RNAs and precision run-on sequencing during mouse erythropoiesis.
[Molecular Cell]
Reimer, K. A., Mimoso, C. A., Adelman, K., & Neugebauer, K. M. (2021). Co-transcriptional splicing regulates 3′ end cleavage during mammalian erythropoiesis. Molecular Cell, 0(0). https://doi.org/10.1016/j.molcel.2020.12.018 Cite
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The Adverse Events of Hematopoietic Stem Cell Transplantation Are Associated with Gene Polymorphism within Human Leukocyte Antigen Region

Scientists investigated the correlation between the gene polymorphisms within the HLA region and the adverse reactions of post-hematopoietic stem cell transplantation in patients with acute leukemia, where 72 patients were diagnosed with acute lymphocytic leukemia and 104 were acute myeloid leukemia.
[Scientific Reports]
Chen, D.-P., Wen, Y.-H., Wang, P.-N., Hour, A.-L., Lin, W.-T., Hsu, F.-P., & Wang, W.-T. (2021). The adverse events of haematopoietic stem cell transplantation are associated with gene polymorphism within human leukocyte antigen region. Scientific Reports, 11(1), 1475. https://doi.org/10.1038/s41598-020-79369-w Cite
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Chaperone-Mediated Autophagy Sustains Hematopoietic Stem-Cell Function

Scientists showed that chaperone-mediated autophagy, a selective form of lysosomal protein degradation, is involved in sustaining hematopoietic stem cell function in adult mice.
[Nature]
Dong, S., Wang, Q., Kao, Y.-R., Diaz, A., Tasset, I., Kaushik, S., Thiruthuvanathan, V., Zintiridou, A., Nieves, E., Dzieciatkowska, M., Reisz, J. A., Gavathiotis, E., D’Alessandro, A., Will, B., & Cuervo, A. M. (2021). Chaperone-mediated autophagy sustains haematopoietic stem-cell function. Nature, 1–7. https://doi.org/10.1038/s41586-020-03129-z Cite
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Successful Gene Therapy of Diamond-Blackfan Anemia in a Mouse Model and Human CD34+ Cord Blood Hematopoietic Stem Cells Using a Clinically Applicable Lentiviral Vector

Researchers observed that transduced ribosomal protein S19 (Rps19)-deficient bone marrow cells could reconstitute mice longterm and rescue the bone marrow failure and severe anemia observed in Rps19-deficient mice, with a low risk of mutagenesis and a highly polyclonal insertion site pattern.
[Haematologica]
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