Volume 15.37 | Sep 22

Issue 15.37 September 22, 2014
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Reversal of Diabetes with Insulin-Producing Cells Derived In Vitro from Human Pluripotent Stem Cells
Researchers describe a seven-stage protocol that efficiently converts human embryonic stem cells into insulin-producing cells. Stage 7 cells expressed key markers of mature pancreatic beta cells, including MAFA, and displayed glucose-stimulated insulin secretion similar to that of human islets during static incubations in vitro. [Nat Biotechnol] Abstract | Press Release
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PUBLICATIONS (Ranked by impact factor of the journal)
DOK7 Gene Therapy Benefits Mouse Models of Diseases Characterized by Defects in the Neuromuscular Junction
Scientists show in a mouse model of familial limb-girdle myasthenia that therapeutic administration of an adeno-associated virus vector encoding the human DOK7 gene resulted in an enlargement of neuromuscular junctions and substantial increases in muscle strength and life span. [Science] Abstract | Press Release

Pyrimidoindole Derivatives Are Agonists of Human Hematopoietic Stem Cell Self-Renewal
Investigators identified a family of chemically related small molecules that stimulates the expansion ex vivo of human cord blood cells capable of reconstituting human hematopoiesis for at least six months in immunocompromised mice. [Science] Abstract | Press Release

Cardiac Tissue Slice Transplantation as a Model to Assess Tissue-Engineered Graft Thickness, Survival, and Function
Cardiac tissue slices (CTS) were generated by sectioning mouse hearts expressing firefly luciferase and green fluorescent protein into slices of defined size and thickness using a vibrating blade microtome. Bioluminescence imaging of CTS transplanted onto hearts of immunodeficient mice demonstrated survival of ≤30% of transplanted cells. [Circulation] Abstract

Long Term Evaluation of Mesenchymal Stem Cell Therapy in a Feline Model of Chronic Allergic Asthma
Researchers documented the effects of allogeneic, adipose-derived mesenchymal stem cells (MSCs) on airway inflammation, airway hyperresponsiveness, and remodeling over time and investigate mechanisms by which MSCs alter local and systemic immunologic responses in chronic experimental feline allergic asthma. [Clin Exp Allergy] Abstract

Adenosine Kinase, Glutamine Synthetase and EAAT2 as Gene Therapy Targets for Temporal Lobe Epilepsy
Scientists determined whether adeno-associated viral vector-mediated overexpression of glutamine synthetase or excitatory amino-acid transporter 2 (EAAT2), or expression of microRNA targeting adenosine kinase in hippocampal astrocytes in the rat brain could modulate susceptibility to kainate-induced seizures and neuronal cell loss. [Gene Ther] Abstract

Pre-Transplant Mobilization with Granulocyte Colony Stimulating Factor Improves B Cell Reconstitution by Lentiviral Vector Gene Therapy in SCID-X1 Mice
Researchers hypothesized that mobilization of hematopoietic stem cell by granulocyte colony-stimulating factor should create temporary space in bone marrow niches to improve engraftment and thereby B cell reconstitution. [Hum Gene Ther] Abstract

Clinical Cell Therapy Imaging Using a Perfluorocarbon Tracer and Fluorine-19 MRI
The use of a perfluorocarbon tracer agent for clinical fluorine-19 MRI cell detection is described. This technology was used to detect immunotherapeutic dendritic cells delivered to colorectal adenocarcinoma patients. [Magn Reson Med] Full Article | Press Release

Adenovirus-Mediated siRNA Targeting NOB1 Inhibits Tumor Growth and Enhances Radiosensitivity of Human Papillary Thyroid Carcinoma In Vitro and In Vivo
Researchers investigated the association of NIN1/RPN12 binding protein 1 homolog (NOB1) expression with tumor growth and radiosensitivity of human papillary thyroid carcinoma (PTC). The in vitro and in vivo radiosensitivity of PTC cells was determined by clonogenic cell survival assay and a mouse xenograft model, respectively. [Oncol Rep] Abstract

BACE1 RNA Interference Improves Spatial Memory and Attenuates Aβ Burden in a Streptozotocin-Induced Tau Hyperphosphorylated Rat Model
β-secretase (BACE1) RNAi was used to treat the streptozotocin-induced tau hyperphosphorylation animal models. BACE1 RNAi treatment improved the behavioral ability of animal models and reduced the amount of Aβ1-40 and Aβ1-42, accompanied by decreasing the levels of BACE1 and amyloid precursor protein C-terminal fragment. [Cell Biochem Funct] Abstract

View Data: StemSpan™ SFEM II Serum-Free Medium
Biologic Scaffolds for Regenerative Medicine: Mechanisms of In Vivo Remodeling
The authors review the composition of biologic scaffolds, the methods and recommendations for manufacture, the mechanisms of the biomaterial-host interaction, and the clinical application of this regenerative medicine approach. [Ann Biomed Eng] Abstract

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research field.

Learn more about the new smartphone app for human blood cell frequencies
Study Adds to Cancer-Fighting Promise of Combined Immunotherapy-Radiation Treatment
A study in mice implanted with breast and melanoma cancers adds to a growing body of evidence that highly focused radiation – long thought to suppress immunity – can actually help boost the immune system’s fight against cancer when combined with a new kind of immune-enhancing drug. [Press release from Johns Hopkins Medicine (EurekAlert) discussing research presented at the American Society of Radiation Oncology (ASTRO) 56th Annual Meeting, San Francisco] Press Release

Biothera to Present at European Society of Medical Oncology 2014 Congress
Biothera announced that new data and insights for Imprime PGG, the company’s investigational cancer immunotherapy drug, will be featured in three presentations, including a late-breaking abstract. [Press release from Biothera discussing research to be presented at the European Society of Medical Oncology (ESMO) 2014 Congress, Madrid] Press Release

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STAP Co-Author Offers Yet Another Recipe for Stem Cells
A senior co-author on controversial, and now retracted, stem cell papers has quietly posted new tips on how the research can be replicated. Two papers claiming that stressing the body’s cell could produce embryonic-like stem cells, a process called stimulus triggered acquisition of pluripotency (STAP), were heralded when published in Nature in January but thrashed soon after when problematic images and figures were soon found. [Nature News] Editorial

Bad Bugs Inspire White House Task Force and $20 Million Prize
In the fight against antibiotic-resistant bacteria, the U.S. government is dangling a new incentive: a $20 million prize for a quick diagnostic test to recognize highly resistant infections. The prize is just one in a slew of actions announced by the White House to signal its greater attention to the threat of antibiotic-resistant microbes. [ScienceInsider] Editorial
Cynata Announces Landmark Licensing Deal
Cynata Therapeutics Ltd. announced that it has entered into a license agreement with Cellular Dynamics International. The agreement provides Cynata with nonexclusive worldwide rights to a clinical grade human induced pluripotent stem cell line manufactured by CDI. [Cynata Therapeutics Ltd.] Press Release

Boehringer Ingelheim and CureVac Announce Collaboration to Develop Next Generation Lung Cancer Immunotherapy
Boehringer Ingelheim and CureVac jointly announced an exclusive global license and development collaboration. The new collaboration focuses on CureVac’s CV9202, a novel investigational therapeutic mRNA vaccine in early clinical development for the treatment of lung cancer. [Boehringer Ingelheim GmbH] Press Release

Sutro Biopharma and Merck KGaA of Darmstadt, Germany, to Partner on Development of Antibody Drug Conjugates
Sutro Biopharma and the biopharmaceutical division of Merck KGaA, Darmstadt, Germany, announced a collaboration and license agreement to develop antibody drug conjugates. The antibody is thought to specifically target and deliver the cytotoxic drug to the cancer cells. [Sutro Biopharma] Press Release

Medigene AG: New Research Collaborations and Additional Grant for Immunotherapy TABs
Medigene AG announced that its subsidiary Trianta Immunotherapies GmbH entered two new research collaborations for further development of its immunotherapy platform TABs (T-cell-specific AntiBodies) for the treatment of various types of cancer and autoimmune diseases. [Medigene AG] Press Release

Penn Medicine and Wistar Receive $12 Million NCI Melanoma Grant to Help Fast Track Lab Discoveries into Treatments
Penn Medicine and The Wistar Institute have been awarded a prestigious $12.1 million SPORE grant from the National Cancer Institute (NCI). The five-year Specialized Programs of Research Excellence, or SPORE, grant will fund four new melanoma research projects that aim to translate fundamental laboratory discoveries into new therapeutics to treat melanoma and other skin cancers. [Penn Medicine] Press Release

NW Bio’s Cancer Vaccine Is the First Drug to Be Designated by UK Authorities as a “Promising Innovative Medicine” (PIM)
Northwest Biotherapeutics announced that its DCVax-L is the first product to receive formal designation as a PIM under the new “Early Access to Medicines Scheme” (EAMS) launched in the UK. A PIM is the first step in a two-step process for early access approval under the EAMS. [Northwest Biotherapeutics] Press Release

Inovio Pharmaceuticals Expands HPV Immunotherapy Development to Aerodigestive Cancers
Inovio Pharmaceuticals, Inc. announced it has initiated a Phase I clinical trial in patients with aerodigestive cancers. The trial will evaluate the safety, tolerability, and immunogenicity of INO-3106. This immunotherapy targets human papillomavirus type 6 (HPV-6), which causes most aerodigestive cancers. [Inovio Pharmaceuticals, Inc.]
Press Release

Amgen Submits Biologics License Application for Investigational BiTE® Immunotherapy Blinatumomab
Amgen announced submission of a Biologics License Application to the U.S. Food and Drug Administration seeking approval for its investigational bispecific T cell engager (BiTE®) antibody construct, blinatumomab. [Amgen Inc.] Press Release

Cancer Research Institute to Honor Leaders in Cancer Immunotherapy
The Cancer Research Institute (CRI) announced the winners of the 2014 Oliver R. Grace Award for Distinguished Service in Advancing Cancer Research. CRI will present the award to Bristol-Myers Squibb, for leading scientific advances and research in the field of immuno-oncology. [Cancer Research Institute] Press Release
Prospective Grant of Exclusive License: Therapeutics for the Treatment of Lysosomal Storage Disorders (FR Doc. No: 2014-22210)

National Cancer Institute; Notice of Meeting (FR Doc. No: 2014-22129)
2014 Biological Device Application Approvals
Food and Drug Administration (United States)

Medical Devices; Immunology and Microbiology Devices; Classification of Tryptase Test System (FR Doc. No: 2014-22254)
NEW XXII Congress of the European Society of Gene and Cell Therapy (ESGCT)
October 23-26, 2014
The Hague, The Netherlands

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Head of Cellular and Molecular Therapies Laboratory (NHS Blood & Transplant – Oxford)

NEW Head of Cellular and Molecular Therapies Laboratory (NHS Blood & Transplant – Leeds)

NEW Postdoctoral Position – Novel Mediators of Cardiovascular Disease and Novel Therapeutic Strategies (Monzino Cardiology Center)

Research Technician – Stem Cells and Regenerative Medicine (King’s College London)

PhD Opportunities – Cell Therapies (Cooperative Research Centre for Cell Therapy Manufacturing)

Postdoctoral Fellow – Cardiac Differentiation of Pluripotent Stem Cells (Stanford University)

Postdoctoral Fellow – Cardiac Animal Surgery (Stanford University)

Postdoctoral Position – Transplantation Aspects of Hepatocytes Derived from Human Pluripotent Stem Cells (INSERM UMR 1064)

Research Associate – Gene Therapy Center (University of Massachusetts)

Postdoctoral Fellow – New Combination Immune Therapies for Cancer (Massachusetts General Hospital)

Postdoctoral Position – Scaffolding and Drug Delivery for Regenerative Medicine Applications (Ospedale Niguarda)

Scientist – Pluripotent Stem Cell Media Development, High Throughput Screening (STEMCELL Technologies Inc.)

Scientist – Cell Culture Support Products (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Scientist – Immunology/Cell Separation (STEMCELL Technologies Inc.)

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