Volume 13.22 | Jun 11

Issue 13.22 June 11, 2012
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Molecularly Self-Assembled Nucleic Acid Nanoparticles for Targeted In Vivo siRNA Delivery
Investigators showed that self-assembled DNA tetrahedral nanoparticles with a well-defined size can deliver small interfering RNAs (siRNAs) into cells and silence target genes in tumors. [Nat Nanotechnol]
Abstract | Press Release

Complimentary Wallchart: Human Hematopoietic Progenitors. Request Your Copy Today.

PUBLICATIONS (Ranked by impact factor of the journal)

Direct Reprogramming of Mouse and Human Fibroblasts into Multipotent Neural Stem Cells with a Single Factor
Researchers reported the generation of induced neural stem cells from mouse and human fibroblasts by direct reprogramming with a single factor, Sox2. [Cell Stem Cell] Abstract | Press Release

Contrasting Acute Graft-versus-Host-Disease Effects of Tim-3/Galectin-9 Pathway Blockade Dependent Upon the Presence of Donor Regulatory T Cells
These studies show that whereas the absence of T-cell immunoglobulin mucin-3 (Tim-3)/galectin-9 pathway interactions augments systemic graft-versus-host-disease (GvHD), concurrent donor T-regulatory cell depletion paradoxically and surprisingly inhibits GvHD. [Blood] Abstract

HIF-2α Suppresses p53 to Enhance the Stemness and Regenerative Potential of Human Embryonic Stem Cells
Scientists have uncovered a unique transient reprogramming activity in human embryonic stem cells, the “enhanced stemness” reprogramming where a highly cytoprotective and undifferentiated state is achieved by transiently suppressing p53 activity. They suggest that this transient reprogramming is a form of stem cell altruism that benefits the surrounding tissues during the process of tissue regeneration. [Stem Cells]
Abstract | Press Release

High-Purity Enrichment of Functional Cardiovascular Cells from Human iPS Cells
Researchers developed a highly reproducible method that can simultaneously enrich a large number of cardiomyocytes and cardiac smooth muscle cells and endothelial cells from human induced pluripotent stem (iPS) cells with high purity. [Cardiovasc Res] Abstract

Therapeutic Potential of Transplanted Placental Mesenchymal Stem Cells in Treating Chinese Miniature Pigs with Acute Liver Failure
Investigators aimed to isolate mesenchymal stem cells from human placenta and determine their therapeutic potential for treating Chinese experimental miniature pigs with acute liver failure. [BMC Med] Abstract

CXCR4 Gene Transfer Prevents Pressure Overload Induced Heart Failure
Researchers used gene therapy to test the effects of chemokine receptor-4 (CXCR4) gene delivery on adverse ventricular remodeling due to pressure overload. [J Mol Cell Cardiol] Abstract

Intravitreal Delivery of AAV-NDI1 Provides Functional Benefit in a Murine Model of Leber Hereditary Optic Neuropathy
Recombinant adenoassociated virus (AAV) serotype 2 expressing the NADH-quinone oxidoreductase gene (NDI1) (AAV-NDI1) was shown to protect retinal ganglion cells in a rotenone-induced murine model of Leber hereditary optic neuropathy. [Eur J Hum Genet] Full Article

Efficacy of Adjuvant Immunotherapy with Cytokine-Induced Killer Cells in Patients with Locally Advanced Gastric Cancer
Researchers aimed to determine the long-term efficacy of adjuvant immunotherapy with autologous cytokine-induced killer cells for locally advanced gastric cancer patients. [Cancer Immunol Immunother] Abstract

Differentiation of Multipotent Vascular Stem Cells Contributes to Vascular Diseases
Findings support a new hypothesis that the differentiation of multipotent vascular stem cells, rather than the de-differentiation of smooth muscle cells, contributes to vascular remodeling and diseases. [Nat Commun]
Abstract | Press Release

Perivascular Stem Cells: A Prospectively Purified Mesenchymal Stem Cell Population for Bone Tissue Engineering
Investigators prospectively purified human perivascular stem cells from adipose tissue and compared their bone-forming capacity with that of traditionally derived stromal vascular fraction. [Stem Cells Transl Med] Abstract | Press Release

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Ixmyelocel-T Shown to Protect Heart from Damage in Murine Model of Heart Failure
Animals treated with ixmyelocel-T demonstrated a reduced mortality compared to control and decreased infarct size and increased survival when compared to a vehicle control group. Ixmyelocel-T therapy is a patient-specific, expanded multicellular therapy comprised of a mixture of cell types cultured from bone marrow mononuclear cells. [Press release from Aastrom Biosciences, Inc. discussing research presented at the 18th Annual International Society for Cellular Therapy (ISCT) Meeting, Seattle] Press Release

Silence Therapeutics Announces Safe and Effective Dose Successfully Identified in Phase I Study of Atu027 and Potential Fund Raising
The open label, single-center, dose-finding Phase I study of Atu027 in subjects with advanced solid cancer was designed to evaluate up to a total of 11 escalating doses of Atu027. Atu027 was very well tolerated and safe up to the 10th dose level. [Press release from Silence Therapeutics plc discussing research presented at the 2012 American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago] Press Release

Positive Phase Ia Data for AGS-009 in Patients with Lupus
Argos Therapeutics Inc. presented positive Phase Ia clinical data for its monoclonal antibody-based therapy, AGS-009, for the treatment of systemic lupus erythematosus (lupus). In addition to being safe and generally well tolerated by patients, AGS-009 demonstrated a dose-proportional shift toward normalization of the genes regulated by its target, human interferon-alpha. [Press release from Argos Therapeutics Inc. discussing research presented at the 2012 European League Against Rheumatism (EULAR) Congress, Berlin] Press Release

Alnylam Presents Data from Its Phase I Extension Study with ALN-VSP, an RNAi Therapeutic for the Treatment of Liver Cancers
Results were announced from a Phase I extension study with ALN-VSP, a systemically delivered RNA interference (RNAi) therapeutic for the treatment of advanced solid tumors with liver involvement. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the 2012 American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago] Press Release

Watch The Video: Performing CFC Assays Using MethoCult™


White House Petition Urging Open Access Reaches 25,000 Signers
A petition urging the White House to expand federal policies requiring free access to taxpayer-funded research papers has gotten the 25,000 signatures needed to trigger a response. [White House, United States]
Press Release

French Research Unions Challenge Plan to Focus Science Funding
The French scientific community has brought legal action against the country’s new government in order to cancel €7.7 billion worth of funding agreements intended for the development of national excellence clusters. [Excellence Initiatives Program, France] Press Release

Controversial Texas ‘Incubator’ Grant Will Undergo Scientific Review
The Cancer Prevention & Research Institute of Texas says that a controversial “incubator” grant awarded to the University of Texas MD Anderson Cancer Center in Houston will be reevaluated by both scientific and business experts. The institute also plans a broad reconsideration of some of its aims and procedures. [Cancer Prevention & Research Institute of Texas, United States] Press Release

Stem Cell Society Singapore Symposium 2012: Stem Cell Based Therapy


Saneron CCEL Therapeutics, Inc. Announces Clinical Supply and Research Collaboration Agreement with Texas Cord Blood Bank
Saneron CCEL Therapeutics, Inc. and the Texas Cord Blood Bank announced a clinical supply and research collaboration agreement to provide mononuclear cells from clinical grade cord blood for use in regenerative medicine research related to potential new Alzheimer’s, ALS and stroke therapies. The Texas Cord Blood Bank is a division of the South Texas Blood & Tissue Center. [South Texas Blood & Tissue Center] Press Release

OncoSec Medical Inc. Enters Into Exclusive Manufacturing Agreement with VGXI Inc. for Production of Interleukin-12 Plasmid DNA
This exclusive agreement is significant for OncoSec. First, it uses VGXI’s patented DNA plasmid manufacturing process. Second, it leverages VGXI’s expertise for large-scale process development and clinical manufacturing for interleukin-12 plasmid DNA for use in OncoSec’s OMS ElectroImmunotherapy program. [OncoSec Medical Inc.] Press Release

AMRI Burlington Contracted as Aseptic Fill Manufacturer for Market Introduction of Excellagen® Wound Care Product
AMRI announced that its Burlington site was contracted by Cardium Therapeutics and its subsidiary, Tissue Repair Company to manufacture sterile pre-filled syringes in support of the U.S. introduction of Excellagen®, a professional-use, formulated collagen-based product for the management of diabetic foot ulcers, pressure ulcers and other dermal wounds. [Cardium Therapeutics] Press Release

Coronado Biosciences Receives Notice of Allowance for First U.S. Patent Covering CNDO-109
Coronado Biosciences, Inc. announced the U.S. Patent and Trademark Office has issued a Notice of Allowance for the foundation U.S. patent application for CNDO-109, licensed by Coronado Biosciences from the University College London Business PLC, for its Natural Killer cell therapy program. [Coronado Biosciences, Inc.]
Press Release

ReproCELL Launches Alzheimer’s Disease Model Based on iPS-Derived Human Neuronal Cells
ReproCELL, Inc. announced that the company will start commercializing human iPS-derived neurons in which an Alzheimer’s disease related gene has been incorporated. [ReproCELL, Inc.] Press Release

Cellular Dynamics Launches MyCell™ Services
Cellular Dynamics International, Inc. announced the launch of its MyCell™ Services. These services include novel induced pluripotent stem (iPS) cell line reprogramming, genetic engineering and differentiation of iPS cells into commercially available iCell® terminal tissue cells. [Cellular Dynamics International, Inc.] Press Release

Tengion Announces Continued Clinical Progress on Key Milestones for Neo-Urinary Conduit™
Tengion, Inc. announced continued clinical progress on key milestones for the ongoing Phase I trial of the Company’s most advanced product candidate, the Neo-Urinary Conduit. [Tengion, Inc.] Press Release

Arrowhead Research Advances ARC-520 into IND-Enabling Studies for the Treatment of Hepatitis B and Provides Guidance on Development Timeline
Arrowhead Research Corporation announced that its hepatitis B virus (HBV) program has completed all internal preclinical requirements and has named a clinical candidate. The candidate, named ARC-520, is an RNA interference therapeutic actively targeted to the liver using the company’s Dynamic Polyconjugate delivery system and includes two siRNA sequences targeting two different regions of the HBV genome. [Arrowhead Research Corporation] Press Release

Alnylam Completes Enrollment in Phase I Clinical Trial and Initiates Phase II Clinical Trial of ALN-TTR02, an RNA Interference Therapeutic Targeting Transthyretin (TTR) for the Treatment of Transthyretin-Mediated Amyloidosis (ATTR)
Alnylam Pharmaceuticals, Inc. announced that it has completed enrollment in its Phase I trial with ALN-TTR02. Alnylam also announced that it has initiated a Phase II trial with ALN-TTR02 aimed at evaluating clinical activity, safety, and tolerability of multiple once-monthly doses of ALN-TTR02 in ATTR patients. [Alnylam Pharmaceuticals, Inc.] Press Release

Preeminent Tissue Engineering Team to Establish Program at Nationwide Children’s Hospital
Breuer and Shinoka were the first in the world to tissue engineer blood vessels and implant them in human infants for repair of congenital heart defects. They currently have US Food and Drug Administration approval to conduct the first U.S human trial to investigate the safety and effectiveness of this method. They and their team will conduct this work at Nationwide Children’s Hospital. [Nationwide Children’s Hospital] Press Release

Mesoblast Provides Corporate Strategy for Product Development to Treat Neurologic Conditions; Exclusive Commercial Rights to Develop Neuroregenerative Dental Pulp Stem Cells
Mesoblast Limited outlined the Company’s strategic plans to develop adult stem cell therapies for neurodegenerative diseases. [Mesoblast Limited] Press Release


Gene Therapy Society Looking to NIH to Revisit Role of the Recombinant DNA Advisory Committee
The principal U.S. professional society representing gene therapists is awaiting word from NIH about whether or not there will be re-examination of the role of its Recombinant DNA Advisory Committee. They aim to limit its longstanding role in clinical trials while continuing its service as a forum for discussing and educating professionals on policy issues. Press Release

Notice of Change in Activity Code for RFA-HG-12-009 “Clinical Sequencing Exploratory Research” from U01 to UM1 (NOT-HG-12-017)

Community Clinical Oncology Program Groups (U10) (RFA-CA-12-007)

Limited Competition: Research Centers in Minority Institutions (RCMI) Translational Research Network (RTRN) (U54) (RFA-MD-12-005)

Developing and Improving Institutional Animal Resources (G20) (RFA-OD-12-008)

National Cancer Institute; Cancellation of Meeting (FR Doc No: 2012-14128)

National Center for Advancing Translational Sciences; Notice of Closed Meeting (FR Doc No: 2012-13844)

National Institute of Diabetes and Digestive and Kidney Diseases; Notice of Closed Meeting (FR Doc No: 2012-13847)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-13727)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-13743)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-13312)

Draft Guidance for Industry: Amendment to Guidance for Industry: Revised Preventive Measures to Reduce the Possible Risk of Transmission of Creutzfeldt-Jakob Disease and Variant Creutzfeldt-Jakob Disease by Blood and Blood Products


European Medicines Agency (European Union)

Management Board Completes Framework for Conflicts of Interests
The European Medicines Agency’s Management Board adopted a new ‘breach of trust procedure’ to deal with cases of incorrect or incomplete declarations of interests of Board members. Press Release


NEW Select Biosciences – Genomics Research Europe
September 4-5, 2012
Frankfurt, Germany

NEW International Society for Cell & Gene Therapy of Cancer (ISCGT) 2012 Conference
October 5-6, 2012
Singapore, Singapore

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.)

Business Analyst – Product Management (STEMSOFT Software, Inc.)

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

Business Development Manager – Stem Cell (SynGen Inc.)

Controller (SynGen Inc.)

Director: Regulatory Affairs (SynGen Inc.)

Manufacturing Manager (SynGen Inc.)

Chief Scientific Officer (SynGen Inc.)

Postdoctoral Fellow – Department of Bioengineering and Barbara Davis Center (University of Colorado at Denver)

Postdoctoral Position – GIGA-Neurosciences (University of Liège)

Postdoctoral Position – Spinal Cord Neural Stem Cells (Institute for Neurosciences of Montpellier)

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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