Volume 15.21 | Jun 2

Issue 15.21 June 2, 2014
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Coaxing iPS Cells to Become More Specialized Prior to Transplantation Cuts Rejection Risk
Researchers have found that coaxing induced pluripotent stem (iPS) cells in the laboratory to become more-specialized progeny cells before transplantation into mice allows them to be tolerated by the body’s immune system. [Press release from the Stanford School of Medicine discussing online prepublication in Nature Communications] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Generation of a New Therapeutic Peptide that Depletes Myeloid-Derived Suppressor Cells in Tumor-Bearing Mice
Researchers adapted a competitive peptide phage display platform to identify candidate peptides binding myeloid-derived suppressor cells (MDSCs) specifically and generated peptide-Fc fusion proteins (peptibodies). In multiple tumor models, intravenous peptibody injection completely depleted blood, splenic and intratumoral MDSCs in tumor-bearing mice without affecting proinflammatory immune cell types, such as dendritic cells. [Nat Med] Abstract | Press Release

Photoactivation of Endogenous Latent Transforming Growth Factor-β1 Directs Dental Stem Cell Differentiation for Regeneration
Scientists showed that non-ionizing, low-power laser treatment can be used as a minimally invasive tool to activate an endogenous latent growth factor complex, transforming growth factor-β1, that subsequently differentiates host stem cells to promote tissue regeneration. [Sci Transl Med] Abstract | Press Release

Improved Survival with T Cell Clonotype Stability after Anti-CTLA-4 Treatment in Cancer Patients
Cytotoxic T lymphocyte-associated antigen-4 (CTLA-4) blockade in patients with metastatic castration-resistant prostate cancer and metastatic melanoma resulted in both expansion and loss of T cell clonotypes, consistent with a global turnover of the T cell repertoire. [Sci Transl Med] Abstract | Press Release

Transplanted Terminally Differentiated Induced Pluripotent Stem Cells Are Accepted by Immune Mechanisms Similar to Self-Tolerance
Scientists showed in murine models that autologous induced pluripotent stem cell-derived endothelial cells elicit an immune response that resembles the one against a comparable somatic cell, the aortic endothelial cell. [Nat Commun] Abstract | Press Release

In Utero Depletion of Fetal Hematopoietic Stem Cells Improves Engraftment after Neonatal Transplantation in Mice
Scientists used an antibody against the murine c-Kit receptor (ACK2) to deplete fetal host hematopoietic stem cells and increase space within the hematopoietic niche for donor cell engraftment. Fetal mice were injected with ACK2 on E13.5-14.5 and surviving pups were transplanted with congenic hematopoietic cells on day of life one. [Blood] Abstract

Effect of HMGB1 on the Paracrine Action of EPC Promotes Post-Ischemic Neovascularization in Mice
In vivo studies showed hPB-endothelial progenitor cells (EPCs) transplantation improved neurobehavioral outcomes, reduced brain atrophy volume and enhanced neovascularization in transient middle cerebral artery occlusion mice. [Stem Cells] Abstract

Dual-Regulated Lentiviral Vector for Gene Therapy of X-Linked Chronic Granulomatosis
X-linked chronic granulomatous disease mice transplanted with transduced hematopoietic stem cell restored gp91phox expression, and myeloid-specific promoter-driven vectors maintained regulation during BM development. [Mol Ther] Abstract

Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington’s Disease
Scientists examined the merits of administering a recombinant adeno-associated viral vector designed to deliver small interfering RNA that targets the degradation of the huntingtin transcript. [Hum Gene Ther]
Abstract | Press Release

Therapeutic Effect of Bone Marrow Mesenchymal Stem Cells on Laser-Induced Retinal Injury in Mice
Researchers investigated the therapeutic potential of bone marrow mesenchymal stem cells by systemic transplantation in a laser-induced retinal injury model. [Int J Mol Sci] Abstract | Download Full Article

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Advances in Mesenchymal Stem Cell-Based Strategies for Cartilage Repair and Regeneration
The authors discuss the emerging role of mesenchymal stem cells in cartilage regeneration and the most recent advances in development of stem cell-based therapeutics for cartilage regeneration. [Stem Cells Rev] Abstract

Mesenchymal Stem Cell Therapy for Immune-Modulation: The Donor, the Recipient, and the Drugs In-Between
The authors highlight and discuss a few factors that they believe are critical in terms of therapeutic success employing cultured mesenchymal stem cells. [Exp Dermatol] Abstract | Full Article

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

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BrainStorm to Present Phase IIa ALS Clinical Trial Data
BrainStorm Cell Therapeutics, a developer of adult stem cell technologies for neurodegenerative diseases, will be presenting initial results from its Phase IIa ALS trial. [Press release from BrainStorm Cell Therapeutics discussing research to be presented at the Joint Congress of European Neurology, Istanbul] Press Release

Cytonet’s Liver Cell Therapy May Be Effective in Stabilizing Patients with Urea Cycle Disorders Awaiting Liver Transplant
New data on Cytonet’s investigational liver cell therapy found it may help temporarily stabilize pediatric patients with urea cycle disorders while they await liver transplantation. [Press release from BusinessWire discussing research presented at the 2014 Joint Garrod and Canadian Newborn and Child Screening Symposium, Ottawa] Press Release

uniQure Announces Successful Transfection of Liver Cells with AAV5 and Strong Safety Data from Acute Intermittent Porphyria Clinical Trial
uniQure N.V. announced successful transfection of liver cells with the porphobilinogen deaminase gene from an ongoing acute intermittent porphyria dose-escalation Phase I trial conducted in collaboration with the AIPGENE Consortium, a pan-European collaboration. [Press release from uniQure N.V. discussing research presented at the 17th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington] Press Release

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Researcher Behind Stem Cell Controversy Agrees to Retraction
After steadfastly defending her work against accusations of falsified data and an official misconduct ruling, the lead author on two controversial stem cell papers published in Nature has reportedly agreed to retract one of them. [ScienceInsider] Editorial

Tests of Embryonic Stem Cell Treatment Back on Track
A potential treatment for spinal cord injury that made headlines in 2010 as the first human embryonic stem cell therapy to be tested in humans is pushing ahead after a stalled clinical trial and a change of ownership. [ScienceInsider] Editorial

E.U. Commission Rejects Plea to Block Stem Cell Research Funding
The European Commission turned down a request by pro-life organizations to block E.U. funding for research using embryonic stem cells – causing many scientists to breathe a sigh of relief. The commission says the existing rules under the European Union’s science program, Horizon 2020, are appropriate and will not change. [ScienceInsider] Editorial
Sangamo BioSciences and City of Hope Granted Strategic Partnership Award from California Institute for Regenerative Medicine to Support Clinical Trial of Stem-Cell Based ZFP Therapeutic® for HIV/AIDS
Sangamo BioSciences, Inc. announced that the California Institute for Regenerative Medicine has granted a $5.6 million Strategic Partnership Award to fund clinical studies at City of Hope to develop a potentially curative ZFP Therapeutic for HIV/AIDS based on the application of Sangamo’s zinc finger nuclease genome-editing technology in hematopoietic stem/progenitor cells. [Sangamo BioSciences, Inc.] Press Release

Aastrom Completes Acquisition of Sanofi’s Cell Therapy and Regenerative Medicine Business
Aastrom Biosciences, Inc. announced that the company has completed its acquisition of Sanofi’s cell therapy and regenerative medicine business unit. [Aastrom Biosciences, Inc.] Press Release

Stem Cell Agency Invests $32 Million Toward Therapies for Spinal Cord Injury and HIV/AIDS
A promising treatment for patients with spinal cord injuries and a therapy aimed at helping HIV patients create an AIDS-resistant blood system were approved by the state’s stem cell agency, the California Institute for Regenerative Medicine. [California Institute for Regenerative Medicine] Press Release

Maryland Stem Cell Research Commission Funds 31 New Research Proposals in FY 2014 with Continued Focus on Contributing to Cures for Debilitating Diseases and Conditions
The board of directors of the Maryland Technology Development Corporation approved the Maryland Stem Cell Research Commission’s recommendation to fund 31 new proposals with the Maryland Stem Cell Research Fund’s $10.4 million FY2014 budget. [Maryland Stem Cell Research Commission] Press Release

A Spanish Device Produces Packaged Batches of Stem Cells for Regenerative Medicine
The Spanish start-up Aglaris Cell is close to launching onto the market the world’s first bioreactor that cultures cell in a fully automated way, without using toxic additives. [ScienceDaily, LLC] Press Release

Tekmira Initiating Phase I/II Clinical Trial in Patients with Hepatocellular Carcinoma
Tekmira Pharmaceuticals Corporation announced they have met all regulatory requirements to initiate a Phase I/II clinical trial of TKM-PLK1 in patients with Hepatocellular Carcinoma. [Tekmira Pharmaceuticals Corporation]
Press Release

Mesoblast Accelerates Plans for Commercialization Manufacturing Operations in Singapore
Regenerative medicine company Mesoblast Limited announced that it is bringing forward plans for its commercial manufacturing operations in line with anticipated product launches in the United States and other major markets. [Mesoblast Limited] Download Press Release

Prima’s CVac Patent Application Receives US Notice of Allowance Prima BioMed Ltd.
Prima BioMed Ltd. announced that the company received a Notice of Allowance from the United States Patent and Trademark Office for Patent Application 11/561,204 “Compositions for immunotherapy and uses thereof” which protects its CVac™ technology; currently in development for ovarian and pancreatic cancer. [Prima Biomed Ltd.] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR. Doc. No: 2014-12490)
Food and Drug Administration (United States)

Guidance for Industry on Expedited Programs for Serious Conditions-Drugs and Biologics; Availability (FR. Doc. No: 2014-12534)

Medical Devices; Immunology and Microbiology Devices; Classification of Dengue Virus Serological Reagents (FR. Doc. No: 2014-12545)

Draft Guidance for Industry on Best Practices in Developing Proprietary Names for Drugs; Availability (FR. Doc. No: 2014-12348)
NEW FEBS-EMBO 2014 Conference
August 30 – September 4, 2014
Paris, France

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Fellow – Prostate Cancer Research (Henry Ford Hospital)

NEW Cell Biologists – T Cell-Based Therapies (Adaptimmune)

NEW Postdoctoral/Clinical Pharmacist – Hematopoiesis and Laboratory for Cell Therapy (Sanquin)

Postdoctoral Position – Neurotoxicity and Epigenetics (Thomas Jefferson University)

Research/Review Scientist – Cell Therapy and Regenerative Medicine (Center for Biologics Evaluation and Research)

Postdoctoral Position – Cell Death and Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Position – Role of Calreticulin in TGF-Beta Signaling in the Kidney Proximal Tubule under Diabetic Conditions (University of Alabama)

Postdoctoral Fellow – Transgene Immunity in AAV Vector-Based Antiviral and/or Gene Therapeutics (University of Massachusetts)

Postdoctoral Fellow – Neurophysiology of Addiction (Medical University of South Carolina)

Principal Investigator – Molecular Mechanisms of Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Associate – Hematopoietic Differentiation (Northwestern University Feinberg School of Medicine)

Developmental-Stem Cell Biologist (Mayo Clinic – Rochester)

Postdoctoral Fellowship – Hematology, Immunology, Cancer Therapeutics (Nationwide Children’s Hospital)

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

Research Technologist – hPSC (STEMCELL Technologies Inc.)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

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

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

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