Cell Therapy News Volume 21.07 | Mar 2 2020

TOP STORY

Gene Therapy Conversion of Striatal Astrocytes into GABAergic Neurons in Mouse Models of Huntington’s Disease

Researchers report an in vivo cell conversion technology to reprogram striatal astrocytes into GABAergic neurons in both R6/2 and YAC128 Huntington’s disease mouse models through AAV-mediated ectopic expression of NeuroD1 and Dlx2 transcription factors. [Nat Commun] Full Article | Press Release

(Ranked by impact factor of the journal)

CD28/4-1BB CD123 T Cells in Blastic Plasmacytoid Dendritic Cell Neoplasm

In vivo, CD28/4-1BB CD123 CAR T cell therapy displayed strong efficacy by promoting a decrease of blastic plasmacytoid dendritic cell neoplasm blast burden. [Leukemia] Abstract

Mesenchymal Stromal Cells Mediated Delivery of Photoactive Nanoparticles Inhibits Osteosarcoma Growth In Vitro and in a Murine In Vivo Ectopic Model

Scientists tested the efficacy of the photoactivation of mesenchymal stromal cells loaded with nanoparticles in vitro and in a murine in vivo ectopic osteosarcoma model. [J Exp Clin Cancer Res] Abstract

Mesenchymal Stem Cells Promote the Resolution of Cardiac Inflammation after Ischemia Reperfusion via Enhancing Efferocytosis of Neutrophils

Mesenchymal stem cells infused immediately and 24 hours later both markedly ameliorated myocardial ischemia-reperfusion injury, and immediate infusion had more significant outcome. These improvements were associated with neutrophil infiltration, measured by fluorescence-activated cell sorting analysis and immunofluorescence staining. [J Am Heart Assoc] Full Article

Comparison of Experimental Peri-Implantitis Models after Application of Ex Vivo BMP2 Gene Therapy Using Periodontal Ligament Stem Cells

Researchers found that the inferior regenerative outcome of an immediate peri-implantitis model at BMP2 gene therapy using autologous periodontal ligament stem cell transplantation resulted from the defect depths, not the model itself. [Sci Rep] Full Article

Generation of GGTA1^-/-β2M^-/-CIITA^-/- Pigs Using CRISPR/Cas9 Technology to Alleviate Xenogeneic Immune Reactions

Scientists demonstrated that elimination of GGTA1, β2M, and CIITA genes in pigs could effectively alleviate xenogeneic immune responses and prolong pig organ survival in xenogenesis. [Transplantation] Abstract

Long-Term Expansion, Genomic Stability and In Vivo Safety of Adult Human Pancreas Organoids

Human pancreas organoids (hPOs) expanded for months in vitro maintained their ductal morphology, biomarker expression and chromosomal integrity. Xenografts of hPOs survived long-term in vivo when transplanted into the pancreas of immunodeficient mice. Mouse orthotopic transplants showed no signs of tumorigenicity. [BMC Dev Biol] Full Article

Adipose Derived Stem Cells and Platelet Rich Plasma Improve the Tissue Integration and Angiogenesis of Biodegradable Scaffolds for Soft Tissue Regeneration

The authors investigate the biocompatibility of nanocomposite scaffolds to deliver adipose derived stem cells with and without the addition of growth factors using platelet rich plasma in vivo. [Mol Biol Rep] Full Article

Chitosan-Based Thermosensitive Scaffold Loaded with Bone Marrow Derived Mesenchymal Stem Cells Promotes the Motor Function Recovery in Spinal Cord Injured Mice

A new thermosensitive composite hydrogel based on chitosan, hydroxyethyl cellulose, collagen, and β-phosphoglycerate was developed to encapsulate murine bone marrow-derived MSCs for improving the therapeutic efficacy of spinal cord injury mice. [NPJ Regen Med] Abstract

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Multi-Target Approaches to CNS Repair: Olfactory Mucosa-Derived Cells and Heparan Sulfates

Scientists discuss the state of human olfactory cell transplantation as a therapy, considering both its current clinical status and its limitations. Furthermore, they introduce a mesenchymal stromal cell derived from human olfactory tissue, which has the potential to induce multifaceted reparative effects in the environment within and surrounding the lesion. [Nat Rev Neurol] Abstract

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Tessa Therapeutics Announces US FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Its CD30 CAR-T Cell Therapy for the Treatment of Relapsed or Refractory CD30-Positive Classical Hodgkin Lymphoma

Tessa Therapeutics announced that the company’s investigational CD30-directed autologous chimeric antigen receptor T cell (CD30 CAR-T) therapy has been granted Regenerative Medicine Advanced Therapy designation by the FDA for the treatment of patients with relapsed or refractory CD30- positive classical Hodgkin lymphoma. [Tessa Therapeutics] Press Release

European Medicines Agency Grants PRIME and Advanced Therapy Medicinal Product Designations to Janssen’s RPGR Gene Therapy for X-Linked Retinitis Pigmentosa

The Janssen Pharmaceutical Companies of Johnson & Johnson announced that the European Medicines Agency has granted both PRIME (PRIority MEdicines) and Advanced Therapy Medicinal Product designations to the company’s adeno-associated virus-RPGR gene therapy product for the treatment of inherited retinal disease X-linked retinitis pigmentosa. [The Janssen Pharmaceutical Companies of Johnson & Johnson] Press Release

The Parker Institute for Cancer Immunotherapy Awards $2.75 Million to Support Six Early Career Researchers from Leading Research Institutions

The Parker Institute for Cancer Immunotherapy announced six early career researchers awards through the Parker Scholars, Parker Bridge Fellows and Parker Senior Fellows programs. [The Parker Institute for Cancer Immunotherapy] Press Release

Scientists Grapple with US Restrictions on Fetal Tissue Research

The Trump administration’s changes to policy involving material donated from abortions have led scientists to adjust their research projects or seek alternative sources of funding. [The Scientist] Editorial

China Bans Cash Rewards for Publishing Papers

Chinese institutions have been told to stop paying researchers bonuses for publishing in journals, as part of a new national policy to cut perverse incentives that encourage scientists to publish lots of papers rather than focus on high-impact work. [Nature News] Editorial

NEW Advanced Therapies in Neurodegeneration: Bridging Neuroscience, Bioengineering and Optogenetics
April 29-30, 2020
Lisbon, Portugal

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NEW Sr Associate Scientist – Autoimmunity and Tumor Immunotherapy (Amgen)

Research Associate – Stem Cell and Gene Therapy Program (Fred Hutchinson Cancer Research Center)

Postdoctoral Researcher – Cell Therapies for Cancer (Technical University of Denmark)

Postdoctoral Fellow – Inflammation and Cancer (University Hospital of Erlangen)

Lecturer – Clinical Pharmacology (University of Bristol)

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

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

Postdoctoral Fellow – Cancer Research (BC Cancer)

Research Assistant – Cancer Research (BC Cancer)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer)

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