Cell Therapy News Volume 21.03 | Jan 27 2020

TOP STORY

Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease

Researchers report the initial results of nine severely affected X-linked chronic granulomatous disease patients who received ex vivo autologous CD34⁺ hematopoietic stem and progenitor cell-based lentiviral gene therapy following myeloablative conditioning in first-in-human studies. The primary objectives were to assess the safety and evaluate the efficacy and stability of biochemical and functional reconstitution in the progeny of engrafted cells at 12 months. [Nat Med] Abstract

(Ranked by impact factor of the journal)

Isolation of Satellite Cells and Transplantation into Mice for Lineage Tracing in Muscle

Investigators provide an optimized protocol for muscle stem cell isolation and transplantation into mice to deconvolute heterogeneity within isolated stem cell populations. Viable and pure cell populations were isolated within two hours and could then be used for therapeutic intervention or transplantation to uncover the repopulating and differentiation potential in mice, a physiologically relevant in vivo context. [Nat Protoc] Abstract

Transplantation of Human Mesenchymal Stem Cells Genome-Edited with LEF1 Improves Cardio-Protective Effects in Myocardial Infarction

The LEF1 gene was introduced into an AAVS1 locus known as a safe harbor site on chromosome 19 by CRISPR/Cas9-mediated gene integration in hUCB-MSCs. Transplantation of LEF1/hUCB-MSCs onto the infarction region in the rat model significantly improved overall survival. [Mol Ther Nucleic Acids] Abstract | Full Article

Human Induced Pluripotent Stem Cell-Derived GABAergic Interneuron Transplants Attenuate Neuropathic Pain

The authors showed that spinally transplanted, terminally differentiated human induced pluripotent stem cell-derived GABAergic neurons provided significant, long-term, and safe relief from neuropathic pain induced by peripheral nerve injury in mice. Furthermore, iGABAergic neuron transplants survived long term in the injured spinal cord and showed evidence of synaptic integration. [Pain] Abstract | Editorial

Tailorable Hydrogel Improves Retention and Cardioprotection of Intramyocardial Transplanted Mesenchymal Stem Cells for the Treatment of Acute Myocardial Infarction in Mice

Vitality, proliferation, and migration of co-cultured adipose-derived mesenchymal stem cells were evaluated. In addition, mice were subjected to myocardial infarction and were intramyocardially injected with ADSCs, Col-Tgel, or a combination thereof. [J Am Heart Assoc] Abstract

CXCR2-Modified CAR-T Cells Have Enhanced Trafficking Ability That Improves Treatment of Hepatocellular Carcinoma

Based on natural lymphocyte chemotaxis theory and characteristics of solid tumor microenvironments, scientists explored the possibility of enhancing CAR-T cell trafficking by using chemokine receptors. In a xenograft tumor model, they found that expressing CXCR2 in CAR-T cells could significantly accelerate in vivo trafficking and tumor specific accumulation, and improve anti-tumor effect of these cells. [Eur J Immunol] Abstract

Photobiomodulation Plus Adipose-Derived Stem Cells Improve Healing of Ischemic Infected Wounds in Type 2 Diabetic Rats

Researchers sought to investigate the impact of photobiomodulation and adipose-derived stem cells alone and in combination, on the maturation step of wound healing in an ischemic infected delayed healing wound model in rats with type 2 diabetes mellitus (DM2). They randomly divided 24 adult male rats into four groups. DM2 plus an ischemic delayed healing wound were induced in all rats. [Sci Rep] Full Article

Human Liver Stem Cells Express UGT1A1 and Improve Phenotype of Immunocompromised Crigler Najjar Syndrome Type I Mice

The authors evaluated the therapeutic potential of human liver stem cells (HLSCs) in immune-compromised NOD SCID Gamma/Ugt1^−/− mice, which closely mimicked the pathological manifestations in Crigler Najjar Syndrome type I patients. To assess whether HLSCs expressed UGT1A1, decellularized mouse liver scaffolds were repopulated with these cells. [Sci Rep] Full Article

Phenotypic, Transcriptional and Functional Analysis of Liver Mesenchymal Stromal Cells and Their Immunomodulatory Properties

Given the known immunomodulatory properties of mesenchymal stromal cells (MSCs), investigators hypothesized that liver (L-)MSCs were superior immunomodulators and contributed to liver-mediated tolerance. L-MSC, generated from human liver allograft biopsies were compared to MSCs from adipose tissue and bone marrow. [Liver Transpl] Abstract

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Toward an Optimized Process for Clinical Manufacturing of CAR-Treg Cell Therapy

Major challenges remain for developing a standardized, robust, and reliable good manufacturing practice (GMP)-compliant manufacturing process for CAR-Treg cells. The authors review current progress in the field and recommend ways to improve current CAR-Treg manufacturing processes based on lessons learned from first-generation Treg therapeutics as well as from anticancer CAR-T cell development. [Trends Biotechnol] Abstract

CRISPR-Based Synthetic Transcription Factors In Vivo: The Future of Therapeutic Cellular Programming

Better understanding of the design principle controlling gene expression together with technologies that enable spatiotemporal control of transcriptional engineering are fundamental for rational design, improved efficacy, and ultimately safe translation to humans. In this review, scientist will discuss recent advances and integrative strategies that can help pave the path toward a new class of transcriptional therapeutics. [Cell Syst] Abstract

Rabbit ATG/ATLG in Preventing Graft-versus-Host Disease after Allogeneic Stem Cell Transplantation: Consensus-Based Recommendations by an International Expert Panel

A comprehensive review of articles released up to October, 2018 was performed as a source of scientific evidence. Fourteen clinically relevant key questions to the domains indication, administration, and post-transplant management were developed and recommendations were produced using the Delphi technique involving a Panel of 14 experts. [Bone Marrow Transplant] Abstract

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

Celularity Announces FDA Clearance of Landmark IND for CYNK-001, an Allogeneic, Off-the-Shelf Cryopreserved NK Cell Therapy

Celularity, Inc. announced the FDA has cleared the company’s Investigational New Drug (IND) Application for CYNK-001 in patients with glioblastoma multiforme. The clinical investigation of CYNK-001 in patients with GBM is expected to be the first clinical trial in the US to investigate intratumoral administration of an allogeneic NK cell therapy. [Celularity, Inc. (BusinessWire, Inc.)] Press Release

Genprex Receives US FDA Fast Track Designation for Gene Therapy That Targets Lung Cancer

Genprex, Inc. announced that the FDA has granted Fast Track Designation for Genprex’s Oncoprex™ immunogene therapy in combination with EGFR inhibitor osimertinib for the treatment of non-small cell lung cancer (NSCLC) patients with EFGR mutations that progressed after treatment with osimertinib alone. [Genprex, Inc.] Press Release

Krystal Biotech Breaks Ground on Second Commercial Gene Therapy Manufacturing Facility

Krystal Biotech, Inc. announced the ground breaking of the second commercial gene therapy facility in Findlay Township, Pennsylvania. The Findlay-based Current Good Manufacturing Practic facility, named ASTRA, will have the capacity to produce commercial gene therapy medicines to treat patients suffering from debilitating rare diseases. [Krystal Biotech, Inc.] Press Release

Brazil’s Pick of a Creationist to Lead Its Higher Education Agency Rattles Scientists

The appointment of a creationism advocate to lead the agency that oversees Brazil’s graduate study programs has scientists there concerned-yet again-about the encroachment of religion on science and education policy. President Jair Bolsonaro’s administration on Saturday January 25^th named Benedito Guimarães Aguiar Neto to head the agency, known as CAPES, who previously served as the rector for a private religious school which advocates the teaching and study of intelligent design. [ScienceInsider] Editorial

US Officials Revisit Rules for Disclosing Risky Disease Experiments

US disease researchers are pushing the government to be more transparent about federally funded research that involves making pathogens more deadly or more transmissible. Several disease researchers who attended a recent meeting to discuss transparency around such studies say the US government should offer a public explanation when it approves such ‘gain-of-function’ experiments, disclose who made the decision to fund them and make a broad public announcement when a study begins. [Nature News] Editorial

Wuhan Scientists: What It’s Like to Be on Lockdown

Chinese authorities have closed off travel into and out of the virus-hit city of Wuhan in an attempt to stop the outbreak’s spread. Nature spoke to three researchers about what it’s like to be inside Wuhan right now – and talked to one who is trying to get back in. [Nature News] Editorial

Animal Rights Conflict Prompts Leading Researcher to Leave Germany for China

A prominent neuroscientist whose German lab was targeted by animal rights activists is heading to China, where he says he will be freer to pursue his work on macaques and other monkeys. Nikos Logothetis, a director at the Max Planck Institute for Biological Cybernetics in Tübingen, Germany, told colleagues last week that the first members of his lab would move in the coming months to a new International Center for Primate Brain Research in Shanghai. [ScienceInsider] Editorial

FDA

Clinical Drug Interaction Studies – Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions Guidance for Industry (FR Doc. No:2020-01064) Notice

NEW 27^th International Molecular Medicine Tri-Conference
March 1-4, 2020
San Francisco, California, United States

Visit our events page to see a complete list of events in the community.

NEW Lecturer – Clinical Pharmacology (University of Bristol)

Research Assistant – Organoid Modeling for Stem Cell Therapies (Boston Children’s Hospital)

Research Fellow – Regenerative Medicine (University of Edinburgh)

Postdoctoral Researcher – Personalized Cancer Treatments (National University of Ireland Galway)

Postdoctoral Researcher – Drug Delivery (University of Minnesota Twin Cities)

Postdoctoral Researchers – Cell Biology (Medicinal Bioconvergence Research Center)

Postdoctoral Scientist – Functional Pharmacology (Genentech, Inc.)

Postdoctoral Fellow – Cancer Research (BC Cancer)

Research Assistant – Cancer Research (BC Cancer)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer)

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