Volume 14.25 | Jul 1

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TOP STORY
UCLA Stem Cell Gene Therapy for Sickle Cell Disease Advances Toward Clinical Trials
Researchers have successfully established the foundation for using hematopoietic stem cells from the bone marrow of patients with sickle cell disease to treat the disease. They introduced an anti-sickling gene into the hematopoietic stem cells to capitalize on the self-renewing potential of stem cells and create a continual source of healthy red blood cells that do not sickle. [Press release from UCLA discussing online prepublication in the Journal of Clinical Investigation] Press Release | Full Article
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PUBLICATIONS (Ranked by impact factor of the journal)
Acquired Resistance to Anti-VEGF Therapy in Glioblastoma Is Associated with a Mesenchymal Transition
The authors aimed to identify mechanisms responsible for glioblastoma resistance to antiangiogenic therapy. Glioma stem cell NSC11 and U87 cell lines with acquired resistance to bevacizumab were developed from orthotopic xenografts in nude mice treated with bevacizumab. Genome wide analyses were used to identify changes in tumor subtype and specific factors associated with resistance. [Clin Cancer Res] Abstract

β-Mannosylceramide Activates Type I Natural Killer T Cells to Induce Tumor Immunity without Inducing Long-Term Functional Anergy
Researchers determined whether β-mannosylceramide induces anergy of type I natural killer T (iNKT) cells. Induction of anergy was determined by ex vivo analysis of splenocytes from mice pre-treated with iNKT cell agonists as well as in the CT26 lung metastasis in vivo tumor model. [Clin Cancer Res] Abstract

5′-Triphosphate-siRNA against Survivin Gene Induces Interferon Production and Inhibits Proliferation of Lung Cancer Cells In Vitro
Investigators designed and generated 5′-triphosphate-siRNA targeting human survivin gene (3p-survivin-siRNA). They demonstrated that 3p-survivin-siRNA induced a 3p-dependent type-I interferon response when transfected into human lung cancer cells. The 3p-survivin-siRNA significantly inhibited lung cancer cell proliferation in a 3p-dependent manner. [J Immunother] Abstract

Hematopoietic Stem Cell Transplantation with Umbilical Cord Multipotent Stromal Cell Infusion for the Treatment of Aplastic Anemia- A Single-Center Experience
The authors observed the outcome of co-transfusion of umbilical cord multipotent stromal cells and allogeneic hematopoietic stem cells in the treatment of heavily-transfused patients with severe aplastic anemia. [Cytotherapy] Abstract

Blocking Transforming Growth Factor-β Signaling Pathway Augments Antitumor Effect of Adoptive NK-92 Cell Therapy
Scientists aimed to develop a treatment strategy through adoptive transfer of transforming growth factor-β (TGF-β) insensitive NK-92 cells. To block TGF-β signaling pathway, NK-92 cells were genetically modified with dominant negative TGF-β type II receptor by optimizing electroporation using the Amaxa Nucleofector system. These genetically modified NK-92 cells were insensitive to TGF-β and able to resist the suppressive effect of TGF-β on Calu-6 lung cancer cells in vitro. To determine the antitumor activity in vivo, recipient mice were challenged with a single subcutaneous injection of Calu-6 cells. [Int Immunopharmacol] Abstract

Adipose-Derived Stem Cells Combined with Inorganic Bovine Bone in Calvarial Bone Healing in Type 2 Diabetic Rats
The authors investigated whether adipose-derived stem cells (ASCs) combined with inorganic bovine bone improve bone repair in calvarial vertical critical-sized defects (CSD) in type 2 diabetic rats. Bovine bone alone or seeded with 3×105, 3×106 or 3×107 ASCs/graft was randomly transplanted into calvarial CSDs in diabetic rats induced by a high-fat diet with low-dose streptozotocin. [J Peridontol] Abstract Abstract

Pancreatic Tumor Mass in a Xenograft Mouse Model Is Decreased by Treatment with Therapeutic Stem Cells following Introduction of Therapeutic Genes
Scientists employed two types of therapeutic stem cells expressing cytosine deaminase with or without human interferon-β (IFN‑β) to selectively treat pancreatic cancer. [Oncol Rep] Abstract

Transplantation of Bone Marrow Stromal Cells Enhances Nerve Regeneration of the Corticospinal Tract and Improves Recovery of Neurological Functions in a Collagenase-Induced Rat Model of Intracerebral Hemorrhage
Researchers hypothesized that functional restoration after intracerebral hemorrhage (ICH) involves brain reorganization which may be favorably modulated by stem cell transplantation. Here, bone marrow stromal cells were transplanted into the perilesional sites of collagenase induced ICH in adult rats one day after ICH injury. [Mol Cells] Abstract

Brain Food: NeuroCult SM1 for Long-Term Primary Neuronal Culture. View Data!
REVIEWS
Engineering In Vitro Microenvironments for Cell Based Therapies and Drug Discovery
Growing evidence suggests that surface topography, substrate stiffness, mechanical stimulation, oxygen tension and localized density influence cellular functions and longevity, enhance tissue-specific extracellular matrix deposition and direct stem cell differentiation. Researchers discuss how these cues will facilitate engineering of physiological in vitro microenvironments to enable clinical translation of cell based therapies and development of in vitro models for drug discovery applications. [Drug Discov Today] Abstract

The Functions and Applications of RGD in Tumor Therapy and Tissue Engineering
The authors review the main functions and advantages of Arginine-Glycine-Aspartic (RGD), describe the applications of RGD in imaging agents, drugs, gene delivery for tumor therapy, and highlights the role of RGD in promoting the development of tissue engineering (bone regeneration, cornea repair, artificial neovascularization) in recent years. [Int J Mol Sci] Abstract

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

Achieve Superior Differentiation of Mouse MSC & MEF into Osteoblasts with the New MesenCult™ Osteogenic Stimulatory Kit (Mouse)
SCIENCE NEWS
Alnylam Reports Positive Phase II Data for ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR)
Alnylam Pharmaceuticals, Inc. announced the achievement of positive clinical results from its Phase II trial of ALN-TTR02, an RNAi therapeutic targeting the TTR gene for the treatment of ATTR. Interim results show that multiple doses of ALN-TTR02 led to robust and statistically significant knockdown of serum TTR protein levels of up to 93%. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the 2013 Biennial Meeting of the Peripheral Nerve Society, St. Malo] Press Release

Mesoblast Limited Receives Clearance to Begin Phase II Clinical Trial of Mesenchymal Precursor Cells in Diabetic Nephropathy
Mesoblast Limited announced that it has received approvals from Australian ethics committees to commence a Phase II trial evaluating a single intravenous infusion of its proprietary allogeneic or “off-the-shelf” adult mesenchymal precursor cells in patients with type 2 diabetes and advanced kidney disease, or diabetic nephropathy. [Mesoblast Limited] Press Release

The European Cancer Congress 2013
POLICY
First Clinical Trial with Induced Pluripotent Stem Cells Grows Closer
A Japan Ministry of Health, Labour and Welfare panel has accepted a plan to carry out what would be the world’s first clinical trials involving induced pluripotent stem (iPS) cells. The acceptance paves the way for an official green light from the ministry, which could come in early July. Researchers at the RIKEN Center for Developmental Biology in Kobe plan to generate replacement retinal pigment epithelial cells from iPS cells generated from patients suffering age-related macular degeneration.[Japan Ministry of Health, Japan]
Press Release


UK Science Budget Stays Frozen
The United Kingdom (UK) budget for science will remain in the deep freeze, the chancellor of the exchequer George Osborne revealed. But with cuts being made to nearly all sectors except health and schools, some researchers are relieved that science has emerged relatively unscathed. The comprehensive spending review unveiled sets the budgets of all government departments for the 2015-16 financial year and leaves science funding at £4.6 billion – the same level that it has been at since 2010. [UK Government, United Kingdom]
Press Release


E.U. Leaders Agree on Science Budget
European Union (E.U.) leaders have reached a last-minute agreement on the bloc’s budget for the next seven years, which would include €70.2 billion for its flagship research program, Horizon 2020. This is a 23% increase over the previous incarnation, known as Framework Programme 7, which started in 2007 and ends this year. [European Commission, Europe] Press Release

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BUSINESS
Pluristem Enters Into Strategic Partnership and Licensing Agreement With Cha Bio&Diostech for the Development and Commercialization of PLX Cells for the Treatment of CLI and IC in South Korea
Pluristem Therapeutics Inc. announced that its wholly owned subsidiary, Pluristem Ltd., has entered into an exclusive out-license and strategic partnership agreement with Cha Bio&Diostech for the use of Pluristem’s PLacental eXpanded (PLX) cells for peripheral artery disease, specifically in two indications: the treatment of Critical Limb Ischemia (CLI), and Intermediate Claudication (IC) in South Korea. [Pluristem Therapeutics Inc.]
Press Release


Immunocore and Genentech Enter Strategic Alliance to Develop ImmTACs for Multiple Cancer Targets
Immunocore Limited, the Oxford-based biotechnology company developing novel biological drugs known as ImmTACs to treat cancer and viral disease, announced that it has entered into a research collaboration and licensing agreement with Genentech, a member of the Roche Group for the discovery and development of multiple novel cancer targets using Immunocore’s ImmTAC technology. [Immunocore Limited] Press Release

Dr. Robert Langer, Pioneer in Drug Delivery Systems and Regenerative Medicine Joins CHS Pharma, Inc. as a Member of its Board of Directors and Scientific Advisory Board
CHS Pharma Inc. has announced the addition of Robert S. Langer, Sc.D., to its Board of Directors and Scientific Advisory Board. Langer has made pioneering contributions in drug delivery and regenerative medicine, and together with Judah Folkman, M.D., the founder of the field of angiogenesis, isolated the first substances that stopped blood vessels from growing which led to the first treatment for wet age-related macular degeneration and many forms of cancer. [CHS Pharma Inc.] Press Release

BioBridge Global Announced as Holding Company Name for South Texas Blood & Tissue Center and Affiliates
BioBridge Global was announced as the name of the non-profit holding company that includes South Texas Blood & Tissue Center (STBTC), QualTex Laboratories and GenCure. The new structure and corporate name reflect the organization’s evolution and expansion in scope in recent years, and signals the Board’s intent that the new company will serve as a means to grow the enterprise, building and leveraging its capabilities in blood, tissue, bone marrow, cord blood, regenerative medicine, research and biologic testing. [South Texas Blood & Tissue Center] Press Release

Sidra to Open First Reprogrammable Cell Therapy Facility in the Region
Sidra Medical and Research Center will open the MENA region’s first reprogrammable cell therapy facility as part of its state-of-the-art hospital and research center. The facility will investigate a novel treatment approach, called reprogrammable cell therapy, where cells are altered to treat or cure disease. [Sidra Medical and Research Center] Press Release

Extracellular Matrix Key to Cell Therapy Success
Successful translation of basic stem cell (SC) research into commercially viable therapeutic applications relies on the ability to manipulate human cells suitable for regenerative medicine under animal product free conditions. The challenge remains to create serum-free systems in which SCs for human therapy can be placed in a microenvironment for optimal growth and differentiation without the risk of introducing viruses and animal-related pathogens. Local scientists are currently researching what may progress this challenge; the extracellular matrix, a dynamic biological support structure around cells of all tissues and organs in the human body. [Science Network Western Australia] Press Release

NIST Announces New Scaffold Reference Material for Tissue Engineering Research
The National Institute of Standards and Technology (NIST) has issued a new reference material-a sort of standardized sample-of cellular scaffolds for use in tissue engineering research. Growing custom replacement tissue-cartilage, bone, blood vessels, possibly even whole organs-is one of the hot research fields in modern medicine. The new NIST reference material is designed as a common ground for research labs, a well-understood, uniform scaffold that can be used as a baseline or control in experiments that measure factors such as cell adhesion and proliferation. [The National Institute of Standards and Technology] Press Release

Waisman Scientists Model Human Disease in Stem Cells
In the laboratories at the Waisman Center at UW-Madison, scientists are growing induced pluripotent stem cells into models of disorders caused by defective nerve cells. The technology depends on work pioneered over the past decade or so by Su-Chun Zhang, a neuroscientist who leads the iPS Core at Waisman, which also produces cells for other investigators on campus. [University of Wisconsin – Madison] Press Release

Colorado State University Veterinarians Study Stem-Cell Therapy for Chronic Hepatitis
Colorado State University veterinarians are for the first time investigating the use of stem-cell therapy to treat chronic liver disease in dogs, a novel approach they hope could lead to successful treatments for dogs and people. In a new study, veterinarians are looking at treatment of chronic hepatitis, or inflammation of the liver, with mesenchymal stem cells derived from excess fat tissue of healthy adult donors. [Colorado State University] Press Release
NIH
NIH to Reduce Significantly the Use of Chimpanzees in Research
The National Institutes of Health (NIH) plans to substantially reduce the use of chimpanzees in NIH-funded biomedical research and designate for retirement most of the chimpanzees it currently owns or supports.
Press Release


Center for Scientific Review; Notice of Closed Meeting (FR Doc No: 2013-15062)

Government-Owned Inventions; Availability for Licensing (FR Doc No: 2013-15204)
REGULATORY
European Medicines Agency (European Union)

European Medicines Agency Recommends Approval of Fourth Advanced Therapy in Europe
 
EVENTS
NEW Frontiers in Stem Cells and Regeneration
September 29 – October 6, 2013
Woods Hole, United States

NEW TERMIS (Tissue Engineering and Regenerative Medicine International Society) America Conference
November 10-13, 2013
Atlanta, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
JOB OPPORTUNITIES
NEW PhD Positions – Tissue Engineering (8 European Research Institutes)

Postdoctoral Fellowship – Organic Synthesis and Medicinal Chemistry (Masaryk University)

Research Scientist/Senior Research Officer – Monoclonal Antibodies (Bioprocessing Technology Institute/A*STAR)

Postdoctoral Fellow/Research Scientist – Tissue Engineering (Institute of Bioengineering and Nanotechnology)

Director of Cell Processing Facility (S L Collins Associates, Inc.)

Postdoctoral Position – Developmental Neurobiology (North Carolina College of Veterinary Medicine)

Senior Postdoctoral Research Position – Molecular Cardiovascular and Renal Research (University of Oklahoma Health Sciences Center)

Postdoctoral Researcher – Hematopoietic Stem Cells (Albert Einstein College of Medicine)


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