Volume 14.47 | Dec 2

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Synthesis and Delivery of Short, Noncoding RNA by B Lymphocytes
Scientists demonstrated that primary B lymphocytes can be genetically programmed with nonviral plasmid DNA for the biogenesis and delivery of antisense sequences (anti-microRNA) against microRNA-150. [Proc Natl Acad Sci USA] Abstract | Press Release
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PUBLICATIONS (Ranked by impact factor of the journal)
Platelet Gene Therapy Corrects the Hemophilic Phenotype in Immunocompromised Hemophilia A Mice Transplanted with Genetically Manipulated Human Cord Blood Stem Cells
Scientists evaluated the feasibility for lentivirus (LV)-mediated human platelet gene therapy of hemophilia A. Human platelet-FVIII (2bF8) expression was introduced by 2bF8LV-mediated transduction of human cord blood CD34+ cells followed by xeno-transplantation into immunocompromised NSG mice or NSG mice in a FVIIInull background. [Blood] Abstract

MSC-Based VEGF Gene Therapy in Rat Myocardial Infarction Model using Facial Amphipathic Bile Acid-Conjugated Polyethyleneimine
Facially amphipathic bile acid-modified polyethyleneimine conjugates were synthesized and used to transfer hypoxia-inducible vascular endothelial growth factor gene (VEGF) in mesenchymal stem cells (MSCs) for the treatment of rat myocardial infarction. [Biomaterials] Abstract

Tissue Engineered Bulking Agent with Adipose-Derived Stem Cells and Silk Fibroin Microspheres for the Treatment of Intrinsic Urethral Sphincter Deficiency
Scientists developed a tissue engineered bulking agent that consisted of adipose-derived stem cells and silk fibroin microspheres to treat stress urinary incontinence caused by severe intrinsic sphincter deficiency. [Biomaterials] Abstract

Donor Mesenchymal Stromal Cells (MSCs) Undergo Variable Cardiac Reprogramming In Vivo and Predominantly Co-Express Cardiac and Stromal Determinants after Experimental Acute Myocardial Infarction
The authors previously showed the emergence of predominantly non-fused murine cells co-expressing cardiac and stromal determinants in co-cultures of murine MSCs and rat embryonic cardiomyocytes. To determine whether a similar phenotype is detectable in vivo in ischemic myocardium, they infused green fluorescence protein-marked MSCs intravenously into wild-type mice in an acute myocardial infarction model generated by ischemia/reperfusion or fixed coronary artery ligation. [Stem Cell Rev] Abstract

Adipose-Derived Stem Cells Exhibit Antioxidative and Antiapoptotic Properties to Rescue Ischemic Acute Kidney Injury in Rats
The authors used an ischemia-reperfusion-induced acute kidney injury rat model. The effects of rescuing acute kidney injury were assessed with regard to different adipose-derived stem cell numbers and various routes of administration compared with sham-operated and phosphate-buffered saline-treated groups. [Plast Reconstr Surg] Abstract

Nanoparticle Delivery of the Bone Morphogenetic Protein 4 Gene to Adipose-Derived Stem Cells Promotes Articular Cartilage Repair In Vitro and In Vivo
Investigators evaluated the effect of poly(lactic-co-glycolic acid) nanoparticles delivering pDC316-BMP4-EGFP plasmid into rabbit adipose-derived stem cells (ADSCs) in vitro and chondrogenesis of the bone morphogenetic protein 4-transfected ADSCs seeded onto poly(L-lactic-co-glycolic acid) scaffold in a rabbit model. [Arthroscopy] Abstract

MicroRNA-23a Is Involved in Tumor Necrosis Factor-α Induced Apoptosis in Mesenchymal Stem Cells and Myocardial Infarction
Using a tumor necrosis factor (TNF)-α-induced bone marrow mesenchymal stem cell (BM-MSC) injury model in vitro and a rat myocardial infarction (MI) model in vivo, scientists showed study that miR-23a was involved in TNF-α-induced BM-MSC apoptosis through regulating caspase-7 and that the injection of BM-MSCs overexpressing miR-23a could improve left ventricular function and reduce infarct size in the rat MI model. [Exp Mol Pathol] Abstract

Characterization of a Biodegradable Coralline Hydroxyapatite/Calcium Carbonate Composite and Its Clinical Implementation
The ability of coralline hydroxyapatite/calcium carbonate (CHACC) to promote conductive osteogenesis was assessed in vitro using human mesenchymal stem cells and in vivo using an immunodeficient mouse model. The clinical performance of CHACC as a bone substitute to fill voids caused by excision of bone tumors was also observed in 16 patients. [Biomed Mat] Abstract | Press Release

Take the "if" out of "diff": Reduce variability when differentiating to hematopoietic cells
Mesenchymal Stromal Cells to Promote Kidney Transplantation Tolerance
The authors review recent evidence on both the beneficial and deleterious effects of mesenchymal stromal cell (MSC) in experimental models of solid organ transplantation as well as first clinical experiences of MSC therapy in kidney transplant recipients. [Curr Opin Organ Transplant] Abstract

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

Your Free Copy of the Nature Neuroscience Poster Neural Stem Cells Awaits'
Tocagen’s Toca 511 Gene Therapy with Toca FC Was Well Tolerated and Showed Antitumor Activity in Patients with High Grade Glioma
Tocagen Inc. announced that interim clinical data from two ongoing Phase I investigational studies showed that Toca 511 administration followed by courses of oral Toca FC was safe and well tolerated in 60 patients treated to date with recurrent high grade glioma. In addition, data were presented showing that in tumor samples taken from some patients following treatment, Toca 511 selectively infected tumor cells and expressed a cytosine deaminase transgene. [Press release from PR Newswire Association LLC discussing research presented at the Society for Neuro-Oncology (SNO) Annual Meeting, San Francisco] Press Release

Celldex Therapeutics’ Rindopepimut Demonstrates Promising Clinical Activity in Patients with EGFRvIII-Positive Recurrent Glioblastoma
Celldex Therapeutics, Inc. reported interim data from its ongoing, exploratory Phase II ReACT study of rindopepimut in recurrent glioblastoma. Rindopepimut is an immunotherapeutic vaccine that targets the tumor specific oncogene EGFRvIII. [Press release from Cell Therapeutics, Inc. discussing research presented at the Society for Neuro-Oncology (SNO) Annual Meeting, San Francisco] Press Release

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23andMe Ordered to Halt Sales of DNA Tests
The US Food and Drug Administration (FDA) has ordered personal-genomics company 23andMe to stop marketing its DNA-testing service. In a warning letter dated November 22, the FDA , says that 23andMe has not provided information showing that its service is safe and effective. [Nature News] Editorial

UK Government’s Overspending Could Hit Science Budget
Leaked documents suggest that the UK research budget may be cut by 2%, or around £200 million, over two years. The revelations came in an internal memo from the Department for Business, Innovation and Skills that lays out a package of measures ministers are said to be discussing to balance the department’s budget. [Nature News] Editorial
Editas Medicine Created to Discover and Develop Novel Class of Genome Editing Therapeutics
Editas Medicine, a genome editing company, announced it has secured a $43 million Series A financing led by Flagship Ventures, Polaris Partners and Third Rock Ventures with participation from Partners Innovation Fund. [Editas Medicine] Press Release

$500,000 Alliance for Cancer Gene Therapy Grant to Fund Breakthrough Brain Cancer Research
Alliance for Cancer Gene Therapy has provided a $500,000 grant to combat glioblastoma, one of the most difficult types of brain cancer to treat. [Vocus PRW Holdings, LLC] Press Release

University of Utah Doctor Performs Historic First Procedure Using New Technique of Retrograde Gene Therapy on a Human Heart
Ernie Lively became the first patient in the world to undergo retrograde gene therapy at University of Utah Hospital, a novel procedure designed to deliver stem cells to the heart to repair damaged muscle and arteries in the most minimally invasive way possible. [University of Utah Health Care] Press Release

NTCELL Clinical Trial: Recruitment of Further Patients Approved
Living Cell Technologies Limited announced that the independent data safety monitoring board has reviewed the safety data from the initial patient in the Phase I/IIa clinical trial of NTCELL and advised that implants for the additional three patients in the trial can proceed. The Phase I/IIa clinical trial is an open label investigation of the safety and clinical effects of NTCELL in four patients who have been diagnosed with Parkinson’s disease for at least five years. [Living Cell Technologies Limited] Press Release

VBL Therapeutics Receives FDA Fast Track Designation for Lead Compound VB-111
VBL Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead oncology drug VB-111, for prolongation of survival in patients with Recurrent Glioblastoma Multiforme (rGBM). VB-111 was already granted Orphan Drug status for GBM in the U.S. and in Europe. VB-111, given as a simple IV infusion, is a novel gene-therapy drug that targets endothelial cells in the tumor vasculature, acting as a “biological knife”. [VBL Therapeutics] Press Release

ATL1102 for Stem Cell Mobilization: Application to Conduct Human Proof of Concept Clinical Trial Submitted
Antisense Therapeutics has advised that it has submitted a clinical trial application to the responsible Human Research Ethics Committee to conduct a Phase I Stem Cell Mobilization Human Proof of Concept trial of ATL1102. [Antisense Therapeutics Limited] Press Release

Recognition for Tissue Engineering Success
Professor Neil Broom, winner of this year’s MacDiarmid Medal from the Royal Society of New Zealand, used skills and techniques honed in metallurgy and applied them to solve complex problems of biological tissue structure. He has developed novel experimental approaches that have led to major improvements in bio-prosthetic heart valve function, new insights into both joint tissue structure and osteoarthritis and the structural and mechanical basis of intervertebral disc prolapse. [The University of Auckland] Press Release

Tiffany Vo Wins Ruth L. Kirschstein National Research Service Award
Rice University bioengineering graduate student Tiffany Vo has been selected for a Ruth L. Kirschstein National Research Service Award to develop and test an injectable-hydrogel scaffold for craniofacial bone tissue regeneration. [Rice University] Press Release

Blue Horizon International Creates Stem Cell Education Broadcast for Public TV
Blue Horizon Stem Cells announced a strategic partnership with “Leading Edge with Jimmy Johnson”, to inform public television audiences on the current status of medical stem cell treatments. [PR Newswire Association LLC] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-28267)

Prospective Grant of Exclusive Patent License: GMCSF-BclxL-Derived Chimeric Therapeutics for Use in Treatment of Cancer, Neutropenia, CNS Injury and Parkinson’s Disease (FR Doc No: 2013-28374)
Approval Letter – IPOL
Food and Drug Administration (United States)

Agency Information Collection Activities; Proposed Collection; Comment Request; Adverse Event Program for Medical Products (FR Doc No: 2013-28600)
NEW Gordon Research Seminar: Craniofacial Morphogenesis & Tissue Regeneration
March 29-April 4, 2014
Lucca, Italy

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Chief Medical Officer (Immunomedics, Inc.)

NEW Postdoctoral Fellow – Wound Healing (University of Connecticut Health Center)

NEW Postdoctoral Fellow – Cardiovascular Tissue Engineering (Stanford University School of Medicine)

Faculty Positions – Stem Cell and Regenerative Medicine (Universidad de Los Andes)

Postdoctoral Fellow – Liver Tissue Engineering (Harvard Medical School)

Research Associate – Stem Cells & Regenerative Medicine (King’s College London)

Assistant Director – Regenerative Medicine Clinical Center (Wake Forest Institute for Regenerative Medicine)

Postdoctoral Positions – Stem Cell, Cancer Biology, Immunology and Bioengineering (UC Irvine)

Faculty Position – Stem Cell Biology (University of Notre Dame)

Postdoctoral Position – Stem Cells Research (University of Maryland Baltimore)

Scientist – Particle Chemistry (STEMCELL Technologies Inc.)

Research Technologist – Human Pluripotent Stem Cell Products (STEMCELL Technologies Inc.)

Research Associate/Scientist – Antibodies Group (STEMCELL Technologies Inc.)

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