Cell Therapy News Volume 21.16 | May 4 2020

TOP STORY

Differentiation of Transplanted Hematopoietic Stem Cells Tracked by Single-Cell Transcriptomic Analysis

Scientists uncovered the previously inaccessible kinetics and fate choices of transplanted hematopoietic stem cells in myeloablated recipients at early stage. [Nat Cell Biol] Abstract

(Ranked by impact factor of the journal)

Engineered Niches Support the Development of Human Dendritic Cells in Humanized Mice

Engraftment of engineered mesenchymal stem cells together with CD34⁺ hematopoietic stem and progenitor cells created an in vivo synthetic niche in the dermis of immunodeficient mice driving the differentiation of classical dendritic cells (DCs) and CD123⁺AXL⁺CD327⁺ pre/AS-DCs. [Nat Commun] Full Article

Cellular Backpacks for Macrophage Immunotherapy

Scientists report an engineered particle referred to as a “backpack” that could robustly adhere to macrophage surfaces and regulate cellular phenotypes in vivo. [Sci Adv] Full Article

Anti-Human CD117 CAR T-Cells Efficiently Eliminate Healthy and Malignant CD117-Expressing Hematopoietic Cells

The authors successfully generated anti-CD117 CAR T-cells from healthy donors and acute myeloid leukemia (AML) patients. Anti-CD117 CAR T-cells efficiently targeted healthy and leukemic CD117-positive cells in vitro. In mice xenografted with healthy human hematopoiesis, they eliminated CD117-expressing, but not CD117-negative human cells. [Leukemia] Abstract

CRISPR/Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich’s Ataxia

Investigators optimized the gene editing approach in hematopoietic stem and progenitor cells isolated from healthy and Friedreich’s ataxia patients’ peripheral blood, and demonstrated normal hematopoiesis of gene-edited cells in vitro and in vivo. [Mol Ther Methods Clin Dev] Abstract | Full Article | Graphical Abstract

A Soluble Endoplasmic Reticulum Factor as Regenerative Therapy for Wolfram Syndrome

Researchers showed that mesencephalic astrocyte-derived neurotrophic factor (MANF), a neurotrophic factor secreted from endoplasmic reticulum (ER) stressed cells, prevented ER stress-mediated β cell death and enhanced β cell proliferation in cell and mouse models of Wolfram syndrome, a prototype of ER disorders. [Lab Invest] Full Article

Human β-Defensin 3 Gene Modification Promotes the Osteogenic Differentiation of Human Periodontal Ligament Cells and Bone Repair in Periodontitis

Investigators found that the human β-defensin 3 gene-modified human periodontal ligament cells presented with high levels of osteogenesis-related gene expression and calcium deposition. [Int J Oral Sci] Full Article

Immunostimulatory Oncolytic Virotherapy for Multiple Myeloma Targeting 4-1BB and/or CD40

Scientists proposed and evaluated immunostimulatory lokon oncolytic adenoviruses for multiple myeloma treatment. [Cancer Gene Ther] Full Article

Microfluidic Synthesis of Injectable Angiogenic Microgels

When encapsulated with cells, angiogenic microgels improved wound healing and liver regeneration, compared with non-angiogenic or acellular microgels. [Cell Rep Phys Sci] Full Article | Graphical Abstract

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Advances in Genome Editing: The Technology of Choice for Precise and Efficient β-Thalassemia Treatment

CRISPR/Cas9 and prime gene editing to correct mutations in hematopoietic stem cells of β-thalassemia patients has been considered a novel therapeutic approach for effective hemoglobin production. However, genome-editing technologies, along with all advantages, have shown some disadvantages due to either random insertions or deletions at the target site of edition or non-specific targeting in genome. [Gene Ther] Abstract

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UM Offers Stem Cells to Treat COVID-19 Patients with Life-Threatening Respiratory Infections

For COVID-19 patients in severe respiratory distress, the University of Miami Miller School of Medicine (UM) now offers access to mesenchymal stem cell treatments that may help fight the characteristic inflammation that accompanies the viral infection. [University of Miami Miller School of Medicine] Press Release

Adverum Biotechnologies Doses First Patient in Cohort 4 of OPTIC Phase I Clinical Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD

Adverum Biotechnologies, Inc. announced that the first patient was dosed in Cohort 4 of the ongoing OPTIC Phase I clinical trial for ADVM-022 for the treatment of wet age-related macular degeneration (AMD). [Adverum Biotechnologies, Inc] Press Release

Editas Medicine Initiates IND-Enabling Activities For EDIT-201, an Allogeneic NK Cell Medicine for the Treatment of Solid Tumor Cancers

Editas Medicine, Inc. announced the initiation of IND-enabling activities for EDIT-201, an allogeneic natural killer (NK) cell medicine for the treatment of solid tumor cancers. [Editas Medicine, Inc.] Press Release

Marker Therapeutics Receives FDA Orphan Drug Designation for its Multi-Antigen Targeted T Cell Therapy for Acute Myeloid Leukemia

Marker Therapeutics, Inc. announced that the FDA Office of Orphan Products Development has granted Orphan Drug designation to MT-401, a multi-tumor-associated antigen-specific T cell product for the treatment of patients with acute myeloid leukemia, following allogeneic stem cell transplant. [Marker Therapeutics, Inc.] Press Release

As COVID-19 Forces Conferences Online, Scientists Discover Upsides of Virtual Format

As the novel coronavirus outbreak shutters businesses and disrupts everyday life for billions around the globe, massive annual conferences and small society meetings alike have moved online. The new format poses numerous technical and organizational challenges, but it also offers opportunities- for reaching wider audiences, reducing the carbon footprint of meeting travel, and improving diversity and equity. [ScienceInsider] Editorial

The Pandemic’s Effects on Recruiting International STEM Trainees

Pandemic-related concerns that have arisen for science trainees—shuttered labs, research and education delays, anxiety—are applicable across the board. But international graduate students and postdocs face a suite of unique challenges that raise uncertainty about recruiting and supporting this critical group of researchers in the future. [The Scientist] Editorial

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NEW Principal Scientist – Human iPSC Gene Engineering (Empyrean Therapeutics Ltd)

Lab Director – Cancer Immunotherapy (Vrije Universiteit Brussel)

Postdoctoral Fellow – Cancer Drug Discovery (Uniformed Services University of Health Sciences)

Scientist – Cancer Immunotherapy (Vrije Universiteit Brussel)

Postdoctoral Associate – Diabetes (University of Miami Health System)

Senior Research Scientist – Orthopedic Surgery/Regenerative Medicine (NYU Grossman School of Medicine)

Team Leaders – Stem Cell Biology (RIKEN Center for Biosystems Dynamics Research)

Postdoctoral Research Associate – Induced Pluripotent Stem Cell Therapies (The University of Arizona College of Medicine)

Professorship – Biological Therapies of the Inner Ear (Hannover Medical School)

Group leaders – Cancer Biology (Institute of Pharmacology and Structural Biology)

Scientist – Cancer Immunotherapy (Lund University)

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