Cell Therapy News Volume 21.14 | Apr 20 2020

TOP STORY

Pharmacologic Fibroblast Reprogramming into Photoreceptors Restores Vision

Transplantation of chemically induced photoreceptor-like cells into the subretinal space of rod degeneration mice led to partial restoration of the pupil reflex and visual function. [Nature] Abstract | Press Release

(Ranked by impact factor of the journal)

Regenerative Metaplastic Clones in COPD Lung Drive Inflammation and Fibrosis

When transplanted to immunodeficient mice, variant clones induced pathology akin to the mucous and squamous metaplasia, neutrophilic inflammation, and fibrosis seen in chronic obstructive pulmonary disease (COPD). [Cell] Abstract | Graphical Abstract

Pooled Knockin Targeting for Genome Engineering of Cellular Immunotherapies

Scientists developed a widely adaptable technology to barcode and track targeted integrations of large non-viral DNA templates and applied it to perform pooled knockin screens in primary human T cells. Pooled knockin of dozens of unique barcoded templates into the T cell receptor-locus revealed gene constructs that enhanced fitness in vitro and in vivo. [Cell] Abstract | Graphical Abstract

AAV-CRISPR Gene Editing Is Negated by Pre-Existing Immunity to Cas9

Investigators tested in a mouse model whether pre-existing immunity to SaCas9 would pose a barrier to liver genome editing with AAV packaging CRISPR/Cas9. [Mol Ther] Abstract | Full Article | Graphical Abstract

MSCs Ameliorate Hepatocellular Apoptosis Mediated by PINK1-Dependent Mitophagy in Liver Ischemia/Reperfusion Injury through AMPKα Activation

Scientists investigated the effect and potential mechanism of mesenchymal stem cells (MSCs) against liver ischemia/reperfusion (I/R) injury. Effects of MSCs were studied in mice liver I/R injury model and in a hypoxia/reoxygenation model of L02 hepatocytes. [Cell Death Dis] Full Article

Stem Cell-Derived Human Striatal Progenitors Innervate Striatal Targets and Alleviate Sensorimotor Deficit in a Rat Model of Huntington Disease

Researchers showed that in vitro differentiated human striatal progenitors underwent maturation and integrated into host circuits upon intra-striatal transplantation in a rat model of Huntington disease. [Stem Cell Reports] Full Article

Novel Drug Candidates Improve Ganglioside Accumulation and Neural Dysfunction in GM1 Gangliosidosis Models with Autophagy Activation

Treatment of the patient-derived neurons with the candidate agents activated autophagy pathways, reducing GM1 ganglioside accumulation in vitro and in vivo, and restoring the presynaptic dysfunction. [Stem Cell Reports] Full Article | Graphical Abstract

Muscle Overexpression of Klf15 via an AAV8-Spc5-12 Construct Does Not Provide Benefits in Spinal Muscular Atrophy Mice

Administration of AAV8-Krüppel-like factor 15 (KLF15) to severe Taiwanese Smn^−/−;SMN2 or intermediate Smn^2B/− spinal muscular atrophy mice significantly increased Klf15 expression in muscle. [Gene Ther] Full Article

Oncolytic Adenovirus Encoding LIGHT (TNFSF14) Inhibits Tumor Growth via Activating Anti-Tumor Immune Responses in 4T1 Mouse Mammary Tumor Model in Immune Competent Syngeneic Mice

Using a subcutaneous model of 4T1 cells in BALB/c mice, rAd.Light was delivered intratumorally to evaluate the anti-tumor responses. Both rAd.Light and rAd.Null significantly inhibited the tumor growth, but rAd.Light produced much stronger anti-tumor effects. [Cancer Gene Ther] Abstract

A Cross-Reactive Small Protein Binding Domain Provides a Model to Study Off-Tumor CAR-T Cell Toxicity

Human CAR-T cells targeted against HER2 using a small protein binding domain induced acute, dose-dependent toxicities in mice. The inclusion of a CD28 or 4-1BB co-stimulatory domain in the CAR was required to produce toxicity, however co-stimulation through CD28 was most toxic on a per-cell basis. [Mol Ther Oncolytics] Abstract | Full Article | Graphical Abstract

An In Vivo Genome-Wide CRISPR Screen Identifies the RNA-Binding Protein Staufen2 as a Key Regulator of Myeloid Leukemia

In a newly developed knockout mouse, loss of staufen2 (Stau2) led to a profound decrease in leukemia growth and improved survival in mouse models of the disease. Further, Stau2 was required for growth of primary human blast crisis chronic myeloid leukemia and acute myeloid leukemia. [Nat Cancer] Abstract

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Current Status of Cell-Based Therapies for Respiratory Virus Infections: Applicability to COVID-19

Limited pre-clinical data suggest that systemic MSC administration can significantly reduce respiratory virus-induced lung injury, however, there are no available data in models of coronavirus respiratory infection. [Eur Respir J] Abstract

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Pluristem Expands Its Compassionate Use Program: Treated First COVID-19 Patient in US under FDA Single Patient Expanded Access Program

Pluristem Therapeutics Inc. announced that it has treated its first patient suffering from COVID-19 complications in the United States under the FDA Single Patient Expanded Access Program. [Pluristem Therapeutics Inc.] Press Release

FDA Authorizes Initiation of a Phase II Trial Evaluating MultiStem^® Cell Therapy in Trauma Patients

Athersys, Inc. announced that the FDA has authorized an Investigational New Drug application to initiate a Phase II clinical trial evaluating Athersys’ MultiStem^® cell therapy for early treatment of traumatic injuries and the subsequent complications that result following severe trauma. [Athersys, Inc.] Press Release

Queen’s University Leading Cell Therapy Clinical Trial to Help Improve Outcomes in COVID-19 Patients

Researchers at Queen’s University Belfast are leading a UK-wide clinical trial, offering an innovative cell therapy treatment for COVID-19 patients with acute respiratory failure. [Queen’s University Belfast] Press Release

Precigen Announces Clearance of IND to Initiate Phase I/II Study for First-in-Class PRGN-2009 AdenoVerse^™ Immunotherapy to Treat HPV-Positive Solid Tumors

Precigen, Inc. announced that the FDA has cleared the Investigational New Drug (IND) application to initiate a Phase I/II trial for Precigen’s PRGN-2009, a first-in-class, off-the-shelf investigational immunotherapy utilizing the AdenoVerse^™ platform designed to activate the immune system to recognize and target HPV⁺ solid tumors. [Precigen, Inc.] Press Release

How Research Funders Are Tackling Coronavirus Disruption

While science is crucial to the fight against COVID-19, researchers confined to their homes and unable to carry on with grant-funded work are becoming increasingly concerned about how the coronavirus pandemic will affect their funding. [Nature News] Editorial

‘Short-Sighted.’ Health Experts Decry Trump’s Freeze on US Funding for WHO as World Fights Pandemic

President Donald Trump said he will suspend US funding to the World Health Organization (WHO) pending a 60- to 90-day investigation into how the agency has handled the response to the COVID-19 pandemic. [ScienceInsider] Editorial

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

NEW Scientist – Cancer Immunotherapy (Vrije Universiteit Brussel)

Postdoctoral Associate – Diabetes (University of Miami Health System)

Associate/Senior Editor – Nature Biotechnology (Nature Research)

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)

Postdoctoral Position – Cancer Immunotherapy (The University of Texas MD Anderson Cancer Center)

Sr Associate Scientist – Autoimmunity and Tumor Immunotherapy (Amgen)

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