Volume 13.24 | Jun 25

Issue 13.24 June 25, 2012
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Blood-Brain Barrier Building Blocks Forged from Human Stem Cells
The blood-brain barrier may be poised to give up some of its secrets as researchers have created, in the laboratory dish, the cells that make up the brain’s protective barrier. The researchers described transforming stem cells into endothelial cells with blood-brain barrier qualities. [Press release from the University of Wisconsin-Madison discussing online prepublication in Nature Biotechnology] Press Release | Abstract

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PUBLICATIONS (Ranked by impact factor of the journal)

Sustained Therapeutic Reversal of Huntington’s Disease by Transient Repression of Huntingtin Synthesis
With antisense oligonucleotides that catalyze RNase H-mediated degradation of huntingtin mRNA, researchers demonstrated that transient infusion into the cerebrospinal fluid of symptomatic Huntington’s disease mouse models not only delays disease progression but mediates a sustained reversal of disease phenotype that persists longer than the huntingtin knockdown. [Neuron] Abstract | Press Release

T Cell Suicide Gene Therapy Prompts Thymic Renewal in Adults after Hematopoietic Stem Cell Transplantation
Data show that the infusion of genetically modified donor T cells after transplantation can drive the recovery of thymic activity in adults, leading to immune reconstitution. [Blood] Abstract

Bioenergetic and Functional Consequences of Cellular Therapy: Activation of Endogenous Cardiovascular Progenitor Cells
Researchers demonstrated that a severely abnormal heterogeneity of myocardial bioenergetics in hearts with postinfarction LV remodeling can be alleviated by human embryonic stem cell-derived vascular cell therapy. [Circ Res] Abstract

Derivation and Propagation of Human Embryonic Stem Cell Lines from Frozen Embryos in an Animal Product-Free Environment
The protocols described are comprehensive instructions for deriving human embryonic stem cell lines in xeno-free conditions from cryopreserved embryos. [Nat Protoc] Abstract

Donor Satellite Cell Engraftment Is Significantly Augmented When the Host Niche Is Preserved and Endogenous Satellite Cells Are Incapacitated
Researchers used the mdx mouse model of Duchenne muscular dystrophy to investigate the effect of the host satellite cell niche on the contribution of donor muscle stem cells (satellite cells) to muscle regeneration. [Stem Cells] Abstract

Production and Implantation of Renal Extracellular Matrix Scaffolds from Porcine Kidneys as a Platform for Renal Bioengineering Investigations
Investigators produced and examined acellular renal extracellular matrix scaffolds as a platform for kidney bioengineering. [Ann Surg] Abstract | Press Release

Transplantation of Engineered Chimeric Liver with Autologous Hepatocytes and Xenobiotic Scaffold
Researchers developed a chimeric liver (CL) by repopulation of rat hepatocytes in a mouse and successfully transplanted it into a rat recipient with vessel reconstruction. This study investigated the feasibility of CL for supporting the recipient after auxiliary liver grafting. [Ann Surg] Abstract

Secondary Sphere Formation Enhances the Functionality of Cardiac Progenitor Cells
When transplanted into the infarcted myocardium, secondary cardiospheres (CSs) engrafted robustly, improved left ventricular dysfunction, and reduced infarct sizes more than sphere-derived cells did. In addition to the cardiovascular differentiation of transplanted secondary CSs, robust vascular endothelial growth factor synthesis and secretion enhanced neovascularization in the infarcted myocardium. [Mol Ther] Abstract

BMP-6 Is More Efficient in Bone Formation than BMP-2 When Overexpressed in Mesenchymal Stem Cells
Previously, mesenchymal stem cells (MSCs) overexpressing bone morphogenetic proteins (BMPs) were shown to differentiate into the osteogenic lineage and induce bone formation. Scientists concluded that bone marrow-derived MSCs nucleofected with rhBMP-6 are superior at inducing bone formation in vivo than all other groups studied. [Gene Ther] Abstract

Albumin-Coated Bioactive Suture for Cell Transplantation
Researchers hypothesized that growing stem cells on the surface of absorbable sutures in vitro and then implanting them via stitching would be a suitable delivery route for cell therapy. Fibronectin, poly-L-lysine, and albumin coatings were used to increase attachment of human and rat bone-marrow-derived mesenchymal stem cells to polyfilament absorbable sutures in vitro. [Surg Innov] Abstract

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Pluristem Therapeutics’ Cell Therapy Broadens Addressable Markets – Demonstrates Systemic Effectiveness of Intramuscular Delivery
The results of the study demonstrated a significant survival and recovery rate of bone marrow and peripheral blood counts in animals pre-irradiated by high lethal doses. These findings indicate that the intramuscular route of administration of placental expanded cells stimulate the hematopoietic stem cells of the bone marrow to produce red and white blood cells as well as platelets crucial for the treatment of hematological disorders. [Press release from Pluristem Therapeutics, Inc. discussing research presented at the 2012 Bio International Convention, Boston] Press Release

Sixteenth Patient Dosed In Neuralstem ALS Stem Cell Trial
Neuralstem, Inc. announced that the first patient to receive stem cell transplantation in both regions of the spinal cord has been treated in the ongoing Phase I trial of its spinal cord neural stem cells in amyotrophic lateral sclerosis (ALS). This is also the 16th patient to be treated in the trial altogether and the first patient returning to the trial for a second treatment. [Neuralstem, Inc.] Press Release

NewLink Updates Enrollment Information for Its Pivotal Phase III HyperAcute(R) Pancreas Immunotherapy (Algenpantucel-L) Trial
NewLink Genetics Corporation announced that it passed the midpoint in enrollment in its Phase III HyperAcute Pancreas immunotherapy product candidate (algenpantucel-L) trial as number of participating institutions reached to 74 sites around the US. [NewLink Genetics Corporation] Press Release

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European Parliament Urged to Continue Funding Research Using Embryonic Stem Cells
A coalition of leading funders of biomedical research and patients groups has issued a joint statement calling on the European Parliament to continue funding research using embryonic stem cells. [Wellcome Trust, United Kingdom] Press Release

U.K. Panel Backs Open Access for All Publicly Funded Research Papers
A long-awaited government report in the United Kingdom says open access journals should be “the main vehicle for the publication of research.” But it warns that such a move would cost the United Kingdom more than $80 million per year in publication charges and other costs. [The Working Group on Expanding Access to Published Research Findings, United Kingdom] Press Release

A Bigger E.U. Research Budget? Don’t Count On It Yet
European researchers should be cautious about their hopes for a big budget boost under Horizon 2020. Financial constraints among E.U. member states could shrink the proposed €80 billion budget to €60 billion or even less. [European Association of Research and Technology Organisations, European Union] Press Release

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New Scottish-Californian Stem Cell Research Announced
Scientists from Heriot-Watt together with their counterparts in St Andrews and Stanford universities are to work together on new medical research that could lead to major breakthroughs for people living with life-threatening conditions. The Heriot-Watt and St Andrews teams will work with research groups at Stanford to examine how stem cells grow and divide and how they change shape to form different types of tissue. [Heriot-Watt University] Press Release

uniQure Collaborates with University of California, San Francisco on GDNF Gene Therapy in Parkinson’s Disease
uniQure announced the signing of a collaborative agreement with two leading neurology experts to develop further a gene therapy incorporating uniQure’s GDNF (glial cell derived neurotrophic factor) gene for the treatment of Parkinson’s disease. [PR Newswire] Press Release

CSHL Announces Collaboration to Speed Development of New Cancer Therapies
Cold Spring Harbor Laboratory (CSHL) has announced a research collaboration with Pfizer Inc. to develop a next-generation human short hairpin RNA library, which could be used to silence gene expression via the process of RNA interference and identify new therapeutic targets in cancer. [Cold Spring Harbor Laboratory]
Press Release

Sistemic Announces Customer Agreement with Cytori Therapeutics
Sistemic Ltd. has formed a customer relationship with Cytori Therapeutics. Cytori will utilize Sistemic’s proprietary SistemQC™  technology platform in its U.S. cardiac cell therapy clinical trial to molecularly characterize adipose-derived stem and regenerative cells. [Sistemic Scotland Limited] Press Release

$5 Million Gift Establishes Gallagher Family Professorships in Adult Stem Cell Research
Alumnus Michael Gallagher and his wife, Elizabeth, have made a $5 million gift to establish the Elizabeth and Michael Gallagher Family Professorships in Adult Stem Cell Research at the University of Notre Dame. [University of Notre Dame] Press Release

Gov. Malloy Announces Award of $9.8 Million in Stem Cell Research Funding
Governor Dannel P. Malloy announced the awards of $9.8 million in stem cell research funds to 19 Connecticut-based researchers. The awards were made by the State of Connecticut Stem Cell Research Advisory Committee at its grant review meeting week. [State of Connecticut, Department of Public Health] Press Release

NeoStem Awarded NIAID Research Grant for the Development of VSEL Technology for Radiation Exposure
NeoStem, Inc. announced that it has been awarded a two year grant totaling $595,252 for the “Development of Human, Autologous, Pluripotent Very Small Embryonic Like (VSELs) Stem Cells as a Countermeasure to Radiation Threat” from the National Institute of Allergy and Infectious Diseases (NIAID), a division of the National Institutes of Health. [NeoStem, Inc.] Press Release

bluebird bio Receives U.S. and European Orphan Drug Designation for Novel Gene Therapy to Treat Adrenoleukodystrophy
bluebird bio announced that both the U.S. Food and Drug Administration and the European Medicines Agency have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy. The product consists of the patient’s own CD34+ hematopoietic stem cells transduced with bluebird bio’s lentiviral vector encoding the human ABCD1 cDNA. [bluebird bioTM] Press Release

Alnylam Receives Orphan Drug Designation from U.S. Food & Drug Administration for ALN-TTR02, an RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis (ATTR)
Alnylam Pharmaceuticals, Inc. announced that the U.S. Food & Drug Administration has provided Orphan Drug Designation to ALN-TTR02 as a therapeutic for the treatment of familial amyloidotic polyneuropathy, one of the predominant clinical manifestations of ATTR. [Alnylam Pharmaceuticals, Inc.] Press Release

Rensselaer and New York State Launch New Stem Cell Research Center
Ground-breaking research to advance the application of stem cells to address critical injuries and diseases will be taking place at Rensselaer Polytechnic Institute, in a new center funded by New York state. [Rensselaer Polytechnic Institute] Press Release

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Frequently Asked Question (FAQs) for RFA-HG-12-006 – Genomic Medicine Pilot Demonstration Pilot Projects (U01) (NOT-HG-12-018)

NIAMS Building Interdisciplinary Research Team (BIRT) Revision Awards (R01) (RFA-AR-13-003)

NIH Support for Conferences and Scientific Meetings (Parent R13/U13) (PA-12-212)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-15150)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-15332)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-15207)


2012 Biological License Application Approvals

Approval History, Letters, Reviews and Related Documents – HPC, Cord Blood

Public Roster of the June 12, 2012 Blood Products Advisory Committee Meeting

Meeting Agenda of the June 12, 2012 Blood Products Advisory Committee Meeting


Food and Drug Administration (United States)

Agency Information Collection Activities; Announcement of Office of Management and Budget Approval; Notice of Participation (FR Doc No: 2012-15130)

Agency Information Collection Activities; Announcement of Office of Management and Budget Approval; Filing Objections and Requests for a Hearing on a Regulation or Order (FR Doc No: 2012-15132)


NEW The Jackson Laboratory: Modeling Human Disease with Diet-Induced Obesity (DIO) Models and Resources
July 10, 1:00PM (ET in US)

NEW 3rd Annual Developing More Predictive In Vitro Models Summit
September 25-27, 2012
Boston, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.


Scientist – Induced Pluripotent Stem Cells (STEMCELL Technologies, Inc.)

Scientist – Epithelial Cell Research (STEMCELL Technologies, Inc.)

Scientist – hPSC Bioengineering (STEMCELL Technologies, Inc.)

Scientist – Mesenchymal Cell Research (STEMCELL Technologies, Inc.)

Research Associate – Chemistry (STEMCELL Technologies, Inc.)

Research Associate – Particle Chemistry (STEMCELL Technologies, Inc.)

Research Technologist – Contract Assay Services (STEMCELL Technologies, Inc.)

Research Technologist – Media Development (STEMCELL Technologies, Inc.)

iPSC Scientist Position – Laboratory of Translational Cell Biology (The Bassell Lab, Emory University)

Chief Scientific Officer (SynGen Inc.)

Cellular Therapy Lab Specialist (The University of Texas Medical School at Houston)

Postdoctoral Research Position for Minority Candidate (New York Obesity Research Center)

Clinical Coordinator (New York Obesity Nutrition Research Center)

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