Targeted Gene Correction of Human Hematopoietic Stem Cells for the Treatment of Wiskott – Aldrich Syndrome

The authors showed that a CRISPR/Cas9-based genome editing strategy allowed the precise correction of Wiskott-Aldrich syndrome mutations in up to 60% of human hematopoietic stem and progenitor cells, without impairing cell viability and differentiation potential.
[Nature Communications]
Rai, R., Romito, M., Rivers, E., Turchiano, G., Blattner, G., Vetharoy, W., Ladon, D., Andrieux, G., Zhang, F., Zinicola, M., Leon-Rico, D., Santilli, G., Thrasher, A. J., & Cavazza, A. (2020). Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome. Nature Communications, 11(1), 4034. https://doi.org/10.1038/s41467-020-17626-2 Cite
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Allogeneic Stem Cell Transplantation for Patients with Aggressive NK-Cell Leukemia

Using the Japanese transplant registry data, the outcomes of 59 aggressive NK-cell leukemia patients who underwent first allogeneic hematopoietic stem cell transplantation were analyzed. Twenty-nine patients received stem cells from cord blood, 18 from peripheral blood, and 12 from bone marrow.
[Bone Marrow Transplantation]
Fujimoto, A., Ishida, F., Izutsu, K., Yamasaki, S., Chihara, D., Suzumiya, J., Mitsui, T., Doki, N., Sakai, H., Kobayashi, H., Kanda, J., Fukuda, T., Atsuta, Y., & Suzuki, R. (2020). Allogeneic stem cell transplantation for patients with aggressive NK-cell leukemia. Bone Marrow Transplantation, 1–10. https://doi.org/10.1038/s41409-020-01009-8 Cite
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Resolving Fates and Single-Cell Transcriptomes of Hematopoietic Stem Cell Clones by PolyloxExpress Barcoding

Scientists found that differentiation-inactive, multilineage, and lineage-restricted hematopoietic stem cell clones reside in distinct regions of the transcriptional landscape of hematopoiesis.
[Cell Stem Cell]
Pei, W., Shang, F., Wang, X., Fanti, A.-K., Greco, A., Busch, K., Klapproth, K., Zhang, Q., Quedenau, C., Sauer, S., Feyerabend, T. B., Höfer, T., & Rodewald, H.-R. (2020). Resolving Fates and Single-Cell Transcriptomes of Hematopoietic Stem Cell Clones by PolyloxExpress Barcoding. Cell Stem Cell, 0(0). https://doi.org/10.1016/j.stem.2020.07.018 Cite
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The Polycomb-Associated Factor PHF19 Controls Hematopoietic Stem Cell State and Differentiation

Researchers showed that, in stark contrast to results published for other Polycomb repressive complex 2 subunits, genetic depletion of Phf19 increases hematopoietic stem cell identity and quiescence.
[Science Advances]
Vizán, P., Gutiérrez, A., Espejo, I., García-Montolio, M., Lange, M., Carretero, A., Lafzi, A., Andrés-Aguayo, L. de, Blanco, E., Thambyrajah, R., Graf, T., Heyn, H., Bigas, A., & Croce, L. D. (2020). The Polycomb-associated factor PHF19 controls hematopoietic stem cell state and differentiation. Science Advances, 6(32), eabb2745. https://doi.org/10.1126/sciadv.abb2745 Cite
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Activated HoxB4-induced Hematopoietic Stem Cells from Murine Pluripotent Stem Cells via Long-Term Programming

Researchers describe the role of HoxB4 in indirectly inhibiting the emergence of mature CD45+ hematopoietic stem cells from induced pluripotent stem cells in vitro.
[Experimental Hematology]
Izawa, K., Yamazaki, S., Becker, H. J., Bhadury, J., Kakegawa, T., Sakaguchi, M., & Tojo, A. (2020). Activated HoxB4-induced Hematopoietic Stem Cells from Murine Pluripotent Stem Cells via Long-Term Programming. Experimental Hematology, 0(0). https://doi.org/10.1016/j.exphem.2020.08.003 Cite
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Acute Alcohol Intoxication Impairs SHH‐Gli1 Signaling and Activation of Primitive Hematopoietic Precursor Cells in the Early Stage of Host Response to Bacteremia

Investigators determined the effect of alcohol exposure on sonic hedgehog signaling in the regulation of hematopoietic stem cell activation during bacteremia.
[Alcoholism-Clinical and Experimental Research]
Shi, X., Simms, K. J., & Zhang, P. (n.d.). Acute Alcohol Intoxication Impairs SHH-Gli1 Signaling and Activation of Primitive Hematopoietic Precursor Cells in the Early Stage of Host Response to Bacteremia. Alcoholism: Clinical and Experimental Research, n/a(n/a). https://doi.org/10.1111/acer.14429 Cite
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Celularity Announces the Activation of First California Clinical Trial Site following CIRM Grant Award to Advance Treatments for COVID-19

Celularity announced that it has been awarded a $750,000 COVID-19 Project grant by the California Institute for Regenerative Medicine (CIRM), one of the three clinical awards targeting the coronavirus. This grant will support California Institutions participating in the Phase I/II clinical trial of human placental hematopoietic stem cell derived natural killer cells for the treatment of adults with COVID-19.
[Celularity Inc.]
Press Release

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A Phase I Study to Evaluate Two Doses of Wharton’s Jelly-Derived Mesenchymal Stromal Cells for the Treatment of De Novo High-Risk or Steroid-Refractory Acute Graft Versus Host Disease

A two-dose cohort trial allogeneic Wharton’s Jelly-derived mesenchymal stromal cells were tested in ten patients with de novo high risk or steroid refractory acute graft versus host disease post allogeneic hematopoietic stem cell transplantation.
[Stem Cell Reviews and Reports]
Soder, R. P., Dawn, B., Weiss, M. L., Dunavin, N., Weir, S., Mitchell, J., Li, M., Shune, L., Singh, A. K., Ganguly, S., Morrison, M., Abdelhakim, H., Godwin, A. K., Abhyankar, S., & McGuirk, J. (2020). A Phase I Study to Evaluate Two Doses of Wharton’s Jelly-Derived Mesenchymal Stromal Cells for the Treatment of De Novo High-Risk or Steroid-Refractory Acute Graft Versus Host Disease. Stem Cell Reviews and Reports. https://doi.org/10.1007/s12015-020-10015-8 Cite
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Hematopoietic Stem Cell Transplantation and Chimeric Antigen Receptor T Cell for Relapsed or Refractory Diffuse Large B-Cell Lymphoma

Scientists treated 14 relapsed or refractory diffuse large B-cell lymphoma patients by combining autologous hematopoietic stem cell transplantation and anti-CD19 chimeric antigen receptor T-cell therapy.
[Immunotherapy]
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Prognostic Impact of TP53 Mutation, Monosomal Karyotype, and Prior Myeloid Disorder in Nonremission Acute Myeloid Leukemia at allo-HSCT

Researchers analyzed prognostic gene mutations by targeted next-generation sequencing to identify predisposing factors for predicting overall survival at one month before transplantation. They enrolled 120 patients with nonremission acute myeloid leukemia who underwent first allogeneic hematopoietic stem cell transplantation (allo-HSCT) between 2005 and 2018.
[Bone Marrow Transplantation]
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Clinical and Molecular Consequences of Fusion Genes in Myeloid Malignancies

The authors discuss the history, biologic effect, and clinical impact of fusion genes in the field of myeloid leukemias.
[Stem Cells]
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Clonal Hematopoiesis and Non-Hematologic Disorders

Researchers review recent studies linking clonal hematopoiesis to altered immune function, inflammation, and non-malignant diseases of aging.
[Blood]
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