Volume 13.37 | Sep 24

Issue 13.37 September 24, 2012
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Feasibility, Safety, and Therapeutic Efficacy of Human Induced Pluripotent Stem Cell-Derived Cardiomyocyte Sheets in a Porcine Ischemic Cardiomyopathy Model
Researchers hypothesized that human induced pluripotent stem cell-derived cardiomyocyte-sheet transplantation would be feasible, safe, and therapeutically effective for the treatment of ischemic cardiomyopathy. [Circulation] Abstract

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PUBLICATIONS (Ranked by impact factor of the journal)

Single-Cell Expression Analyses during Cellular Reprogramming Reveal an Early Stochastic and a Late Hierarchic Phase
During cellular reprogramming, only a small fraction of cells become induced pluripotent stem cells. Previous analyses of gene expression during reprogramming were based on populations of cells, impeding single-cell level identification of reprogramming events. Here, researchers utilized two gene expression technologies to profile 48 genes in single cells at various stages during the reprogramming process. [Cell]
Abstract | Press Release | Graphical Abstract

CXCR1/2 Inhibition Enhances Pancreatic Islet Survival After Transplantation
In humans, the CXCR1/2 allosteric inhibitor reparixin improved outcome in a phase II randomized, open-label pilot study with a single infusion of allogeneic islets. [J Clin Invest] Full Article

Zinc-Finger Nuclease Mediated Correction of α-Thalassemia in iPS Cells
Researchers report the correction of α-thalassemia major hydrops fetalis in transgene-free iPS cells using zinc finger-mediated insertion of a globin transgene in the AAVS1 site on human chromosome 19. [Blood] Abstract

Identification of a Specific Reprogramming-Associated Epigenetic Signature in Human Induced Pluripotent Stem Cells
Researchers characterized the epigenomic integrity of 17 human induced pluripotent stem cell (hiPSC) lines derived from six different cell types with varied reprogramming efficiencies. They demonstrated that epigenetic aberrations are a general feature of the hiPSC state and are independent of the somatic cell source. [Proc Natl Acad Sci USA] Abstract | Press Release

Human Pluripotent Stem Cell-Derived Mesenchymal Stem Cells Prevent Allergic Airway Inflammation in Mice
The authors developed a mouse model of ovalbumin-induced allergic inflammation in both the upper and lower airways and evaluated the effects of the systemic administration of human induced pluripotent stem cell-derived mesenchymal stem cells (MSCs) and bone marrow-derived MSCs on allergic inflammation. [Stem Cells] Abstract

Myoblasts Derived from Normal hESCs and Dystrophic hiPSCs Efficiently Fuse with Existing Muscle Fibers following Transplantation
The authors developed a two-step procedure to differentiate human embryonic stem cells (hESCs) and dystrophic human-induced pluripotent stem cells (hiPSCs) in myogenic cells. When transplanted in the muscle of Rag/mdx mice, these cells participated in muscle regeneration by fusing very well with existing muscle fibers. [Mol Ther] Full Article

Nonviral Delivery of Small Interfering RNA Into Pancreas-Associated Immune Cells Prevents Autoimmune Diabetes
Scientists showed that early (starting at 6-week-old) short-course treatment with lipid/Alox15-specific small interfering RNA complex promoted long-term protection from type 1 diabetes in wild-type nonobese diabetic mice. [Mol Ther] Abstract

Cell-Intrinsic Abrogation of TGF-β Signaling Delays but Does Not Prevent Dysfunction of Self/Tumor-Specific CD8 T Cells in a Murine Model of Autochthonous Prostate Cancer
Researchers used a model of autochthonous murine prostate cancer to evaluate the effect of cell-intrinsic abrogation of TGF-β signaling in self/tumor-specific CD8 T cells used in adoptive T cell therapy to target the tumor in situ. They found that persistence and antitumor activity of adoptively transferred effector T cells deficient in TGF-β signaling were significantly improved in the cancerous prostate. [J Immunol] Abstract

Mesenchymal Stem Cell 1 (MSC1)-Based Therapy Attenuates Tumor Growth Whereas MSC2-Treatment Promotes Tumor Growth and Metastasis
Researchers previously developed a new approach to induce the conventional mixed pool of mesenchymal stem cells (MSCs) into two uniform but distinct phenotypes they termed MSC1 and MSC2. Here they tested the in vitro and in vivo stability of MSC1 and MSC2 phenotypes as well as their effects on tumor growth and spread. [PLoS One] Full Article

Adiponectin Gene Therapy Ameliorates High-Fat, High-Sucrose Diet-Induced Metabolic Perturbations in Mice
Investigators concluded that adiponectin gene therapy ameliorates the metabolic abnormalities caused by feeding mice a high-fat, high-sucrose diet and may be a potential therapeutic strategy to improve obesity-mediated impairments in insulin sensitivity. [Nutr Diabetes] Full Article | Press Release

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Nanomaterials Design and Tests for Neural Tissue Engineering
The purpose of this review is to provide a critical overview of the recent advances in and drawbacks and potential of several fields, contributing to the realization of effective nanotech-based therapies for the regeneration of peripheral nerve transections, spinal cord injuries and brain traumatic injuries. [Chem Soc Rev] Abstract

Bacterial Vectors for Imaging and Cancer Gene Therapy: A Review
This review discusses the currently available imaging technologies and the progress to date exploiting these for monitoring of bacterial gene delivery in vivo. [Cancer Gene Ther] Abstract

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Jennerex Presents Positive Clinical Data from Phase II Trial of JX594 in Sorafenib-Refractory Liver Cancer Patients
Jennerex, Inc. presented Phase II clinical data of JX594 delivered first intravenously and subsequently through intra-tumoral route demonstrating safety as well as disease control and tumor responses in patients with hepatocellular carcinoma. [Press release from Jennerex, Inc. discussing research presented at the International Liver Cancer Association (ILCA) Annual Meeting, Berlin] Press Release

Northwest Bio Proceeding with a Phase I/II Clinical Trial of DCVax®-Direct for All Solid Tumor Cancers
Northwest Biotherapeutics announced that it is in late stage discussions with medical centers in the U.S. and Europe to proceed with a Phase I/II clinical trial with the Company’s third major product line, DCVax®-Direct, for all types of solid tumor cancers. [Northwest Biotherapeutics, Inc.] Press Release

Immunogenicity for Biopharmaceuticals & Biosimilars Asia 2012


China-Japan Territorial Dispute Threatens Scientific Exchanges, Again
A long simmering spat between China and Japan about the sovereignty over a group of uninhabited islets in the East China Sea boiled over again and is, again, denting scientific cooperation. [China Association for Science and Technology, China] Press Release

U.S. Science Advisory Panels Need Greater Transparency, Report Says
U.S. government agencies recruiting scientists and other experts to serve on advisory panels should be asking for more detailed disclosures of potential conflicts of interest. [The Keystone Center, United States]
Press Release

Chilean Scientists Oppose Funding-Agency Relocation
Chilean scientific organizations are alarmed by a suggestion from the country’s economy minister that he should oversee the National Science and Technology Research Commission, the main science funder. [Chilean Economy Minister, Chile] Press Release

Stem Cells 2012 - Mumbia, India


Gallus BioPharmaceuticals and Roslin Cells Announce an Exclusive Partnership to Supply Cell Therapies for Clients
Gallus BioPharmaceuticals, LLC announced a collaboration agreement with Roslin Cells Ltd. to develop and supply clinical and commercial cell therapy products. [Vocus PRW Holdings, LLC.] Press Release

Advanced Cell Technology Secures New $35 Million Funding Commitment from Lincoln Park Capital
Advanced Cell Technology, Inc. announced that it has entered into a $35 million common stock purchase agreement with Lincoln Park Capital Fund, LLC. [Advanced Cell Technology, Inc.] Press Release

CureVac Secures €80 Million in Series D Financing
CureVac GmbH announced the completion of an €80 million financing round with its main investor, dievini Hopp BioTech Holding. In the next years, CureVac will use the proceeds mainly to advance the development of its two lead RNActive® cancer vaccines against prostate cancer and non-small cell lung cancer, respectively. [CureVac GmbH] Press Release

Parent Project Muscular Dystrophy Awards $280,000 for Cardiac Gene Therapy to University of Missouri
Parent Project Muscular Dystrophy announced that it will award the University of Missouri’s Dongsheng Duan, Ph.D. a $280,000 grant to continue his work to treat cardiomyopathy in Duchenne muscular dystrophy through a gene therapy approach. [PR Newswire Association LLC.] Press Release

Gamida Cell Completes Enrollment for Phase I/II Study of Second Pipeline Product Nicord® for Hematological Malignancies
Gamida Cell announced that it has completed enrollment for a Phase I/II clinical trial of NiCord, the company’s second pipeline product. NiCord is an expanded cell graft derived from an entire unit of umbilical cord blood enriched with stem cells. [Gamida Cell] Press Release

NewLink Announces an Investigator Initiated Phase II Study of Sipuleucel-T Plus Indoximod in the Treatment of Certain Men with Advanced Prostate Cancer
NewLink Genetics announced that it will begin an investigator initiated, randomized, double blind placebo controlled Phase II study entitled “Phase II Study of sipuleucel-T (PROVENGE®) plus indoximod (D-1MT/NLG8189) in the treatment of patients with asymptomatic or minimally symptomatic metastatic hormone refractory prostate cancer.” [NewLink Genetics Corporation] Press Release

IntelliCell BioSciences Announces Clinical Study for Diabetic Critical Limb Ischemia
IntelliCell BioSciences, Inc. announced that it will be initiating a groundbreaking in-human clinical study to treat people who suffer from diabetic induced critical limb ischemia wounds. IntelliCell has developed a proprietary treatment methodology using its autologous cellular therapy that includes adult stem cells that have demonstrated regenerative capabilities in anecdotal studies of patients treated. [PR Newswire Association LLC.] Press Release

RBCC Explores NASA Technology in Stem Cell Search
Rainbow Biosciences, the biotech subsidiary of Rainbow Coral Corp. (RBCC), announced that it is exploring the possible use of micro-gravity technology developed by NASA to produce three-dimensional adult stem cells for medical research purposes. [Business Wire] Press Release

Flotte Recognized for Leadership in Biomedical Research
Terence R. Flotte, MD will be honored by the Massachusetts Society for Medical Research for his leadership in gene therapy research and education. [UMass Medical School] Press Release


National Human Genome Research Institute; Notice of Closed Meetings (FR Doc No: 2012-23128)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-23131)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-22927)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-23384)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc No: 2012-23126)


Supporting the Development of Mesenchymal Stem Cells as Medical Therapies

2012 Biological Device Application Approvals


European Medicines Agency (European Union)

Committee for Advanced Therapies’ Activities Streamlined
The European Medicines Agency has replaced the activities of the two working parties of the Committee for Advanced Therapies – the Cell-based Products Working Party and Gene Therapy Working Party – with ad hoc drafting groups. This is part of a drive to improve the efficiency of the Agency’s operations and optimize the use of the expertise available. Press Release

NEW BIT’s 3rd Annual World Congress of Molecular & Cell Biology (CMCB 2013)
June 14-16, 2013
Suzhou, China

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


Scientific Communications & Publishing Coordinator (STEMCELL Technologies, Inc.)

Quality Control Operations Coordinator (STEMCELL Technologies, Inc.)

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

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

Product Manager – Hematopoietic (STEMCELL Technologies, Inc.)

Postdoctoral Fellow – Preclinical Group (BN ImmunoTherapeutics)

Postdoctoral Positions – Immunity and Inflammation (CNRS & University of Toulouse, France)

Postdoctoral Position – Pathophysiology of Brain Tumors (UMR 6301 ISTCT)

Research Associate – Quality Assurance (University of California)

Scientist, Postdoctoral and Research Associate Positions (University of Miami – Sylvester Comprehensive Cancer Center)

Postdoctoral Positions in Cell Biology, Biophysics and Neurobiology (University of Missouri – Kansas City)

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