Volume 14.28 | Jul 22

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TOP STORY
Scientists Transplant Photoreceptors from Retina Grown ‘In a Dish’
Scientists have carried out the first successful transplant of light-sensitive photoreceptor cells extracted from a synthetic retina, grown ‘in a dish’ from embryonic stem cells. When transplanted into night-blind mice these cells appeared to develop normally, integrating into the existing retina and forming the nerve connections needed to transmit visual information to the brain. [Press release from University College London discussing online prepublication in Nature Biotechnology] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
Adeno-associated virus (AAV) vectors delivered through the systemic circulation successfully transduce various target tissues in animal models. However, similar attempts in humans have been hampered by the high prevalence of neutralizing antibodies to AAV, which completely block vector transduction. Researchers showed in both mouse and nonhuman primate models that addition of empty capsid to the final vector formulation can, in a dose-dependent manner, adsorb these antibodies, even at high titers, thus overcoming their inhibitory effect. [Sci Transl Med] Abstract | Press Release

Generation of Functionally Competent and Durable Engineered Blood Vessels from Human Induced Pluripotent Stem Cells
Investigators report a unique approach for the derivation of endothelial precursor cells from human induced pluripotent stem (hiPS) cells using a triple combination of selection markers – CD34, neuropilin 1, and human kinase insert domain-containing receptor – and an efficient 2D culture system for hiPS cell-derived endothelial precursor cell expansion. With these methods, they successfully generated endothelial cells from hiPS cells obtained from healthy donors and formed stable functional blood vessels in vivo, lasting for 280 days in mice. [Proc Natl Acad Sci USA] Abstract | Full Article | Press Release

Self-Organized Vascular Networks from Human Pluripotent Stem Cells in a Synthetic Matrix
Researchers derived a bicellular vascular population from human pluripotent stem cells (hPSCs) that undergoes morphogenesis and assembly in a synthetic matrix. They found that hPSCs can be induced to codifferentiate into early vascular cells (EVCs) in a clinically relevant strategy amenable to multiple hPSC lines. These EVCs can mature into endothelial cells and pericytes, and can self-organize to form microvascular networks in an engineered matrix. These engineered human vascular networks survive implantation, integrate with the host vasculature, and establish blood flow. [Proc Natl Acad Sci USA] Abstract | Press Release

Micro-Dystrophin and Follistatin Co-Delivery Restores Muscle Function in Aged Duchenne Muscular Dystrophy Models
Investigators examined the treatment effect on muscle physiology in aged dystrophic mice with significant disease pathology by combining two promising therapies: micro-dystrophin gene replacement and muscle enhancement with follistatin, a potent myostatin inhibitor. The addition of a muscle enhancement strategy with delivery of follistatin in combination with micro-dystrophin gene therapy completely restored resistance to eccentric contraction induced injury and improved force. [Hum Mol Genet] Abstract

Regenerating Cartilages by Engineered ASCs: Prolonged TGF-β3/BMP-6 Expression Improved Articular Cartilage Formation and Restored Zonal Structure
Scientists employed a baculovirus system that exploited FLPo/Frt-mediated transgene recombination and episomal minicircle formation to genetically engineer rabbit adipose-derived stem cells (rASCs). The baculovirus system conferred prolonged and robust TGF-β3/BMP-6 expression in rASCs cultured in porous scaffolds, which critically augmented rASCs chondrogenesis and suppressed osteogenesis/hypertrophy, leading to the formation of cartilaginous constructs with improved maturity and mechanical properties in two-week culture. Twelve weeks after implantation into full-thickness articular cartilage defects in rabbits, these engineered constructs regenerated neocartilages that resembled native hyaline cartilages in cell morphology, matrix composition and mechanical properties. [Mol Ther] Abstract

Donor Bone Marrow Derived Dendritic Cells Prolong Corneal Allograft Survival and Promote an Intragraft Immunoregulatory Milieu
Investigators describe the ex vivo generation of donor bone marrow derived dendritic cells (BMDCs) and glucocorticoid treated BMDCs with potent immunomodulatory properties for application in allogeneic transplantation. In a fully MHC I/II mismatched rat corneal transplantation model, injection of donor derived untreated BMDC or dexamethasone BMDCs significantly prolonged corneal allograft survival without the need for additional immunosuppression. [Mol Ther] Abstract

Positron Emission Tomography Based In Vivo Imaging of Early Phase Stem Cell Retention after Intramyocardial Delivery in the Mouse Model
The authors aimed to establish PET as a tool for in vivo quantification and monitoring of intramyocardially transplanted stem cells after labeling with FDG in mice with induced myocardial infarction. [Eur J Nucl Med Mol Imaging] Abstract

Gingivae Contain Neural-Crest- and Mesoderm-Derived Mesenchymal Stem Cells
Researchers showed that around 90% of gingivae mesenchymal stem cells (GMSCs) are derived from cranial neural crest cells (CNCC) and 10% from the mesoderm. In comparison with mesoderm MSCs (M-GMSCs), CNCC-derived GMSCs (N-GMSCs) showed an elevated capacity to differentiate into neural cells and chondrocytes and induce activated T-cell apoptosis in vitro. When transplanted into mice with dextran sulfate sodium-induced colitis, N-GMSCs showed superior effects in ameliorating inflammatory-related disease phenotype in comparison with the M-GMSC treatment group. [J Dent Res] Abstract | Press Release

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REVIEWS
Vascular Endothelial Growth Factor in Heart Failure
Researchers outlined the biological characterization of vascular endothelial growth factor (VEGF), and examined the evidence for its potential therapeutic application, including the novel concept of VEGF as adjuvant therapy to stem cell transplantation, in patients with heart failure. [Nat Rev Cardiol] Abstract

Clinical Programs of Stem Cell Therapies for Liver and Pancreas
Regenerative medicine is transitioning into clinical programs utilizing stem/progenitor cell therapies for repair of damaged organs. The authors summarize those for liver and pancreas, organs that share endodermal stem cell populations, biliary tree stem cells, located in peribiliary glands: they are precursors to hepatic stem/progenitors in canals of Hering and to committed progenitors in pancreatic duct glands. [Stem Cells] Abstract

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

Free Wallchart from Nature Neuroscience: Neural Stem Cells
 
SCIENCE NEWS
Advaxis Announces ADXS-cHER2 Demonstrates Significant Survival Advantage in Ongoing Canine Osteosarcoma Study
Advaxis, Inc. announced updated preliminary data from a dose escalation Phase I study evaluating the safety and efficacy of ADXS-cHER2 in the treatment of dogs with osteosarcoma that overexpress human epidermal growth factor receptor-2, or HER2. [Advaxis, Inc.] Press Release

RXi Pharmaceuticals Announces Positive Data in Second Phase I Multi-Dose Study
RXi Pharmaceuticals Corporation announced positive results from their second placebo-controlled Phase I study with RXI 109, an sd-rxRNA® compound that targets Connective Tissue Growth Factor, and is being developed for the treatment of abnormal dermal scars such as hypertrophic scars and keloids in conjunction with scar revision surgery. [RXi Pharmaceuticals Corporation] Press Release

World Cord Blood Congress 2013
 
POLICY
Italian Panel Meets with Controversial Stem Cell Provider to Discuss Trial
A panel of top science administrators and stem cell scientists met with Davide Vannoni, the president of an Italian foundation providing highly controversial stem cell treatments, in a first effort to hash out the details of a government-sponsored clinical trial of the therapy. [Italian Government, Italy] Press Release

Canada’s New Science Minister Lacks Research Pedigree
Former lawyer and nurse Greg Rickford has become Canada’s science minister as Prime Minister Stephen Harper shuffled his Cabinet. It has left science associations scrambling to learn a bit about the new junior minister. [Science Minister, Canada] Press Release

Senate Panel Gives NSF an 8% Budget Boost
In a bipartisan vote, the Senate Appropriations Committee endorsed a 7.9% increase for the agency that would boost its 2014 budget to $7.426 billion. In doing so, it also questioned the value of various initiatives launched by its former director as well as the wisdom of assigning new responsibilities to National Science Foundation (NSF)’s education directorate under a proposed shakeup of all federal science education programs. [National Science Foundation, United States] Press Release

New Law in France Loosens Restrictions on Human Embryo Research
The French National Assembly approved a new law that aims to ease regulation of research involving human embryos and embryonic stem cells. Although French researchers say that the shift will bring little immediate change to their day-to-day work, they hope that the new law will bring more academic freedom and collaboration. [French National Assembly, France] Press Release

U.K. Won’t Eliminate Embryo Research Authority
The U.K. government has decided to keep the country’s Human Fertilisation and Embryology Authority (HFEA) as an independent body overseeing assisted reproduction and embryo research. The government had considered eliminating the agency as part of its push to simplify government and reduce regulation. But the government announced that it would follow the advice of an expert evaluation and leave both HFEA and the Human Tissue Authority in place as independent bodies. [U.K. Government, United Kingdom] Press Release

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BUSINESS
ThermoGenesis and TotipotentRx Announce Definitive Merger Agreement
ThermoGenesis Corp. and TotipotentRx Corporation announced that they have entered into a definitive merger agreement. The combined company is expected to become one of the first fully integrated regenerative medicine companies, developing clinically validated, commercially scalable, point-of-care cell therapies for major therapeutic markets, including orthopedic, cardiovascular and neurologic indications. [ThermoGenesis Corp.] Press Release

Medistem and Cytori Therapeutics Enter into Licensing Agreement
Medistem, Inc. announced that it entered into an agreement granting Cytori Therapeutics, Inc. a license and the exclusive rights to use Medistem’s U.S. Patent No. 8,241,621, “Stem Cell Mediated Treg Activation” in the United States. The licensed patent pertains to Cytori’s Adipose-Derived Regenerative Cell based platform for the treatment of autoimmune diseases. [Medistem, Inc.] Press Release

Mesoblast Receives $4.3 Million from Australian Government for Adult Stem Cell Pipeline Development
Mesoblast Limited announced that it has received approximately $4.3 million under the Australian Government’s Innovation Australia Research and Development (R&D) Tax Incentive Program for R&D activities conducted in Australia during the 2012 financial year. The funds will be used to advance development of Mesoblast’s proprietary Mesenchymal Precursor Cell technology platform and product pipeline. [Mesoblast Limited]
Press Release

ReNeuron Announces £33 Million Financing Package. Placing to Raise Up to £25.35 Million, Grant Package of £7.8 Million from Welsh Government and Notice of General Meeting
ReNeuron announced that it has raised a total of £33 million in a financing package that transforms the Company’s prospects and helps position it as a global leader in stem cell development. [ReNeuron Group plc] Press Release

Audentes Therapeutics Raises $30 Million in Series A Financing
Audentes Therapeutics Inc. announced the closing of a $30 million Series A financing. This funding will allow the Company to further advance its two lead programs, AT001 for X-linked Myotubular Myopathy and AT002 for Pompe disease, and to evaluate additional candidate programs. Both AT001 and AT002 are innovative, novel treatments based on adeno-associated virus gene therapy technology. [Audentes Therapeutics Inc.]
Press Release

STEMSOFT Announces Release of CIBMTR Upload Interface
STEMSOFT Software Inc. announced the release of the new CIBMTR upload interface, STEMSOFTconnect: REGISTRY. This new solution is designed to allow users to complete, submit and review all AGNIS-defined registry forms locally for seamless upload to the Center for International Blood and Marrow Transplant Research (CIBMTR®). [STEMSOFT Software Inc.] Press Release

Information on Proposed Pilot Study of the Safety and Feasibility of Transplantation of Autologous hiPSC-Derived Retinal Pigment Epithelium (RPE) Cell Sheets in Patients with Neovascular Age-Related Macular Degeneration
The RIKEN CDB Laboratory of Retinal Regeneration is currently seeking approval to conduct a small-scale, open label pilot study of the safety of cell sheets incorporating retinal pigment epithelial cells derived from autologous human induced pluripotent stem cells (hiPSCs) in patients with wet-type age-related macular degeneration. [RIKEN Center for Developmental Biology] Press Release

NeoStem Announces Geographic Expansion of Intellectual Property Coverage of AMR-001 to Russia
NeoStem, Inc. announced the geographic expansion of intellectual property protection around its lead product candidate, AMR-001, with the notice of the issuance of patent number 2011127184 in Russia. This patent is in the family titled “Compositions and Methods of Vascular Injury Repair” and protects a chemotactic stem cell product enriched for CD34+ cells that treats injury from acute myocardial infarction. [NeoStem, Inc.]
Press Release  
 
NIH
Notice of Information: NHLBI Will Only Accept Resubmitted Applications for the Extended Expiration Date for PAR-10-234 “Bioengineering Research Partnerships (BRP)(R01)” (NOT-HL-13-183)

Government-Owned Inventions; Availability for Licensing (FR Doc No: 2013-17319)
 
REGULATORY
Food and Drug Administration (United States)

Synergizing Efforts in Standards Development for Cellular Therapies and Regenerative Medicine Products; Public Workshop (FR Doc No: 2013-17528)

Compliance Policy Guide Sec. 253.100–Use of Units of Plasma and Fresh Frozen Plasma Which Have Been Thawed; Withdrawal of Compliance Policy Guide (FR Doc No: 2013-17531)
 
EVENTS
NEW The 5th International Stem Cell Meeting
October 8-9, 2013
Jerusalem, Israel


NEW 2013 Till & McCulloch Meetings
October 23-25, 2013
Banff, Canada


Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
 
JOB OPPORTUNITIES
NEW Postdoctoral Research Associate – Cell Encapsulation for Optimizing Angiogenic Cell Therapies (King’s College London)

Postdoctoral Position – Stem Cell Therapy and Epigenetics of Retina (University of Pittsburgh, Ophthalmology)

Postdoctoral Fellow – Neural Tissue Engineering (School of Chemical & Biomedical Engineering, Nanyang Technological University)

Postdoctoral Fellowship – Immunology (Johns Hopkins University School of Medicine)

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


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