Volume 15.13 | Apr 7

Issue 15.13 April 7, 2014
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Light-Activated Neurons Restore Function to Paralyzed Muscles
A new way to artificially control muscles using light, with the potential to restore function to muscles paralyzed by conditions such as motor neuron disease and spinal cord injury, has been developed. The technique involves transplanting specially-designed motor neurons created from stem cells into injured nerve branches. [Press release from King’s College London discussing online prepublication in Science] Press Release | Abstract | Video
New Cord Banking Software Solution from STEMSOFT
PUBLICATIONS (Ranked by impact factor of the journal)
Prevention and Reversal of Severe Mitochondrial Cardiomyopathy by Gene Therapy in a Mouse Model of Friedreich’s Ataxia
A conditional mouse model with complete frataxin deletion in cardiac and skeletal muscle (Mck-Cre-FxnL3/L- mice) recapitulates most features of Friedreich’s ataxia cardiomyopathy, albeit with a more rapid and severe course. Scientists showed that adeno-associated virus rh10 vector expressing human frataxin injected intravenously in these mice fully prevented the onset of cardiac disease. [Nat Med] Abstract | Press Release

Large-Scale Chondroitin Sulfate Proteoglycan Digestion with Chondroitinase Gene Therapy Leads to Reduced Pathology and Modulates Macrophage Phenotype following Spinal Cord Contusion Injury
Investigators used mammalian-compatible engineered chondroitinase ABC delivered via lentiviral vector to explore the consequences of large-scale chondroitin sulfate proteoglycan digestion for spinal cord repair. [J Neurosci] Abstract | Press Release

Exploitation of Langerhans Cells for In Vivo DNA Vaccine Delivery into the Lymph Nodes
The authors developed a DNA formulation with a polymer and obtained synthetic ‘pathogen-like’ nanoparticles that preferentially targeted Langerhans cells in epidermal cultures. To demonstrate the mechanism of action of this novel vaccination strategy in live animals, they assembled a high-resolution two-photon laser scanning-microscope. [Gene Ther] Abstract

Developing a Potentially Immunologically Inert Tetracycline-Regulatable Viral Vector for Gene Therapy in the Peripheral Nerve
Scientists demonstrated that a chimeric transactivator, created by fusing the Gly-Ala repeat with rtTA and embedded in a lentiviral vector retained its transactivator function in vitro, in muscle explants, and in vivo following injection into the rat peripheral nerve, exhibited a reduced leaky expression, and had an immune-evasive advantage over rtTA as shown in a novel bioassay for human antigen presentation. [Gene Ther] Abstract

Engineering a Micro-Vascular Capillary Bed in a Tissue-Like Collagen Construct
Researchers propose that the coculture of primary endothelial cells with plastic compression collagen constructs, containing a stromal cell population is a valuable technique for in vitro modeling of pro-angiogenic responses towards such bio-mimetic constructs in vivo. [Tissue Eng Part A] Abstract

Sustained Expression of Insulin by a Genetically Engineered Sertoli Cell Line after Allotransplantation in Diabetic BALB/c Mice
Investigators aimed to use a lentiviral vector to achieve stable expression of insulin in Sertoli cells (SCs) and test the ability of these cells to survive after allotransplantation. A mouse SC line transduced with a recombinant lentiviral vector containing furin-modified human proinsulin cDNA, maintained stable insulin expression in vitro. [Biol Reprod] Abstract

A Novel Chimeric Antigen Receptor against PSCA Mediates Tumor Destruction in a Humanized Mouse Model of Pancreatic Cancer
The authors developed a chimeric antigen receptor (CAR) specific for prostate stem cell antigen (PSCA), a glycoprotein that is over-expressed in pancreatic cancer starting at early stages of malignant transformation. To further analyze this CAR, they developed a human pancreatic cancer xenograft model and adoptively transferred CAR engineered T cells into animals with established tumors. [Hum Gene Ther] Abstract

In Vivo Transplantation of Neurosphere-Like Bodies Derived from the Human Postnatal and Adult Enteric Nervous System: A Pilot Study
Investigators transplanted neural progenitor-containing neurosphere-like bodies (NLBs) in a mouse model of hypoganglionosis and analyzed cellular integration of NLB-derived cell types and functional improvement. [PLoS One] Full Article

Adipose-Derived Stromal Cells Inhibit Prostate Cancer Cell Proliferation Inducing Apoptosis
As the effect of adipose-derived stromal cells (AdSCs) on tumor growth has not been studied sufficiently, investigators assessed the degree to which AdSCs affect the proliferation of prostate cancer cells. [Biochem Biophys Res Commun] Abstract

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Stem Cell Transplantation for Amyotrophic Lateral Sclerosis: Therapeutic Potential and Perspectives on Clinical Translation
The authors review the recent advances regarding the therapeutic potential of stem cells for amyotrophic lateral sclerosis with a focus on the minimally invasive strategies that could facilitate an extensive translation to their clinical application. [Cell Mol Life Sci] Abstract

An Update of Human Mesenchymal Stem Cell Biology and Their Clinical Uses
The aim of this review is to revise mesenchymal stem cell biology, their potential translation in therapy, and the challenges facing their adaptation in clinical practice. [Arch Toxicol] Abstract

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

Enter to Win a Travel Award to ISSCR 2014
New Human Trial Shows Stem Cells Are Effective for Failing Hearts
Patients with severe ischemic heart disease and heart failure can benefit from a new treatment in which stem cells found in bone marrow are injected directly into the heart muscle. [Press release from EurekAlert! discussing research presented at the American College of Cardiology’s (ACC) 63rd Annual Scientific Session, Washington, D.C.] Press Release

Threshold Pharmaceuticals Announces Results of Two New Preclinical Studies Evaluating Novel Combinations of TH-302 with Immunotherapy, Antiangiogenic Therapy, and Radiotherapy
Threshold Pharmaceuticals, Inc. announced the results of two new preclinical studies evaluating novel combinations of TH-302, its investigational hypoxia-targeted drug, with immunotherapy, antiangiogenic therapy, and radiotherapy for the potential treatment of cancer. [Press release from Threshold Pharmaceuticals, Inc. discussing research presented at the American Association for Cancer Research (AACR) Annual Meeting 2014, San Diego] Press Release

Attend the 9th Annual World Stem Cells & Regenerative Medicine Congress in London!
European Commission Report Urges Legal Reform to Help Scientists Text-Mine Research Papers
European copyright law should change to help researchers use computer programs to extract facts and data from published research papers, legal experts have urged in a report for the European Commission. [Nature News] Editorial | Report

Former U.S. Research Fraud Chief Speaks Out on Resignation, ‘Frustrations’
David Wright, the director of the federal Office of Research Integrity, which keeps watch on fraud in federally funded biomedical research, quit in frustration after two years. His resignation letter was a scathing critique of what he called the “dysfunctional” bureaucracy at the Office of the Assistant Secretary for Health. [ScienceInsider] Editorial

European Parliament Approves Bill to Increase Clinical Trial Transparency
Researchers who do clinical trials in the European Union will have to make the results public under a bill approved by the European Parliament. In a sweeping vote, 594 members of the Parliament voted in favor of the plan, while only 17 voted against and 13 abstained. [ScienceInsider] Editorial

DARPA Carves Out New Division to Entice Biotech Talent
The U.S. Department of Defense’s research arm is making a concerted grasp at biotechnology. The Defense Advanced Research Projects Agency (DARPA) announced a new division that will consolidate biology research scattered across its existing six divisions and possibly expand the arsenal of projects. [ScienceInsider] Editorial

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Adaptimmune Integrates BioLife Solutions CryoStor® Cell Freeze Media in Cancer Immunotherapy Clinical Trial
BioLife Solutions, Inc. announced that Adaptimmune Ltd has adopted the Company’s CryoStor clinical grade cell freeze media for use in Adaptimmune’s current Phase I/II clinical trial CT Antigen TCR-Engineered T Cells for Myeloma. [BioLife Solutions, Inc.] Press Release

TiGenix Licenses Exclusive Marketing and Distribution Rights for ChondroCelect to Sobi
TiGenix NV announced that it has licensed the marketing and distribution of ChondroCelect, the cell-based medicinal product for the repair of cartilage defects of the knee to Swedish Orphan Biovitrum AB. [TiGenix NV] Download Press Release

Baxter to Acquire Chatham Therapeutics
Baxter International Inc. and Chatham Therapeutics, LLC announced that Baxter has agreed to acquire all of Chatham’s outstanding membership interests. As a result of the transaction, Baxter will acquire Chatham’s developmental gene therapy programs directed toward the development and commercialization of treatments for hemophilia. [Baxter International Inc.] Press Release

Update on Phase III Clinical Trial of Investigational MAGE-A3 Antigen-Specific Cancer Immunotherapeutic in Non-Small Cell Lung Cancer
GlaxoSmithKline plc announced its decision to stop the MAGRIT trial, a Phase III trial of its MAGE-A3 cancer immunotherapeutic in non-small cell lung cancer (NSCLC) patients, after establishing that it will not be possible to identify a sub-population of gene-signature positive NSCLC patients that may benefit from the treatment. [GlaxoSmithKline plc] Press Release

Arrowhead Completes Enrollment of First Cohort in Phase IIa Trial of Chronic Hepatitis B Candidate ARC-520
Arrowhead Research Corporation announced that the first cohort of eight patients has been fully enrolled and dosed in a Phase IIa clinical trial of ARC-520, its RNAi therapeutic for the treatment of chronic hepatitis B virus infection. [Arrowhead Research Corporation] Press Release

FDA Grants IND Approval for Phase IIa Clinical Trial Using CardioCell’s Ischemia-Tolerant Mesenchymal Stem Cell Therapy to Treat Dilated Chronic Heart Failure
CardioCell LLC has received the FDA’s investigational new drug approval for a United States-based, Phase IIa clinical study using its allogeneic stem-cell therapy to treat subjects with chronic heart failure, which generates more than 1 million hospitalizations annually. [CardioCell LLC] Press Release

$2.5 Million Defense Department Grant Funds Gene Therapy Study for Lou Gehrig’s Disease
The Cedars-Sinai Regenerative Medicine Institute has received a $2.5 million grant from the Department of Defense to conduct animal studies that, if successful, could provide the basis for a clinical trial of a gene therapy product for patients with Lou Gehrig’s disease, also called amyotrophic lateral sclerosis, or ALS. [Cedars-Sinai]
Press Release

UTSA Doctoral Students Launch Mobile Stem Care Company that Helps Animals
Two University of Texas at San Antonio (UTSA) biomedical engineering doctoral students, Ramon Coronado and Tony Yuan, have launched Mobile Stem Care LLC, a company that will help veterinarians treat their patients with the latest advancements in stem cell therapies. [The University of Texas at San Antonio] Press Release

Umbilical Cord Stem Cell Therapy Clinical Trial for Multiple Sclerosis Gets Green Light
Translational Biosciences, a subsidiary of Medistem Panama, has received the green light for a Phase I/II clinical trial using human umbilical cord-derived mesenchymal stem cells for multiple sclerosis from the Comité Nacional de Bioética de la Investigación Institutional Review Board in Panama. [Medistem Panama] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc: 2014-07282)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc: 2014-07156)
March 28, 2014 Approval Letter – ALPROLIX
Food and Drug Administration (United States)

Determination of Regulatory Review Period for Purposes of Patent Extension; EYLEA (FR Doc: 2014-07333)
NEW The 2014 Regenerative Medicine Event: Stem Cell Reprogramming
June 2, 2014
London, United Kingdom

NEW Future Investigators of Regenerative Medicine (FIRM) Symposium 2014
September 8-11, 2014
Girona, Spain

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW MSc & PhD Positions – Cell Biology, Neurobiology, Neuroimmunology, Cancer Biology, Computational Biology (Hanyang University)

NEW Postdoctoral Fellow – Therapeutic Corneal Endothelial Cell Culture (Lions VisionGift)

Postdoctoral Research Fellow / Research Officer – Stem Cell Research (Bioprocessing Technology Institute)

Postdoctoral Position – Gene Therapy of Lysosomal Storage Diseases (Cincinnati Children’s Hospital Medical Center)

Cell Therapy Lab Manager (Fred Hutchinson Cancer Research Center)

Stem Cell Research Technician (Van Andel Research Institute)

Postdoctoral Position – Human Stem Cell Biology and Hematopoiesis (Johns Hopkins University School of Medicine)

Director of GMP/GLP Quality Operations (University of Pennsylvania, Perelman School of Medicine)

Cell Culture Specialist – Manufacturing of Cell Therapy Products (Tisch MS Research Center of New York)

Postdoctoral Position – Tissue Engineering and Regenerative Medicine (RUNMC)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

Research Technologist – Pluripotent Stem Cells (STEMCELL Technologies Inc.)

Research Technologist – Particle Chemistry (STEMCELL Technologies Inc.)

Research Technologist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

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