Volume 13.02 | Jan 23

Issue 13.02 January 23, 2012
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AAV6.βARKct Cardiac Gene Therapy Ameliorates Cardiac Function and Normalizes the Catecholaminergic Axis in a Clinically Relevant Large Animal Heart Failure Model
This study was designed to evaluate long-term βARKct expression in a clinically relevant large animal heart failure model, using stable myocardial gene delivery with adeno-associated virus serotype 6 (AAV6). [Eur Heart J] Abstract

See Recommendations: Reducing Immunosuppression Testing By Using Customized Assays To Evaluate Myelotoxicity First

PUBLICATIONS (Ranked by Impact Factor of the Journal)

Embryonic Stem Cell Trials for Macular Degeneration: A Preliminary Report
Researchers’ report provides the first description of human embryonic stem cell-derived cells transplanted into human patients. [Lancet] Abstract | Press Release

Synthetic Mast-Cell Granules as Adjuvants to Promote and Polarize Immunity in Lymph Nodes
Here researchers show that submicrometer particles modeled after mast cell granules augment immunity when used as adjuvants in vaccines. [Nat Mater] Abstract | Press Release

Engraftment of Human HSC in Non-Irradiated Newborn NOD-scidIL2rγnull Mice Is Enhanced by Transgenic Expression of Membrane-Bound Human Stem Cell Factor
Researchers hypothesized that transgenic expression of human membrane-bound stem cell factor would increase levels of human hematopoietic stem cell (HSC) engraftment in non-irradiated NOD-scidIL2rγnull mice and eliminate complications associated with irradiation. [Blood] Abstract

Post-Natal Cardiomyocytes Can Generate iPS Cells with an Enhanced Capacity toward Cardiomyogenic Re-Differentiation
Here, researchers report that neonatal cardiomyocytes can be reprogrammed to generate induced pluripotent stem (iPS) cells that express embryonic-specific markers and feature gene-expression profiles similar to those of mouse embryonic stem cell and cardiac fibroblast-derived iPS cell populations. [Cell Death Differ] Abstract

Nrf3-Pla2g7 Interaction Plays an Essential Role in Smooth Muscle Cell Differentiation from Stem Cells
In this study, embryonic stem cells were cultivated on collagen IV-coated plates to allow smooth muscle cell differentiation. [Arterioscler Thromb Vasc Biol] Abstract

Intranasal Delivery of High Mobility Group Box 1 siRNA Confers Target Gene Knockdown and Robust Neuroprotection in the Postischemic Brain
In the present study, the therapeutic efficacy of intranasal small interfering RNA (siRNA) delivery was investigated in the postischemic rat brain. [Mol Ther] Abstract

Sustained miRNA-Mediated Knockdown of Mutant AAT with Simultaneous Augmentation of Wild-Type AAT Has Minimal Effect on Global Liver miRNA Profiles
Researchers have developed several recombinant adeno-associated virus vectors that incorporate microRNA (miRNA) sequences targeting the a-1 antitrypsin (AAT) gene while also driving the expression of miRNA-resistant wild-type AAT-PiM (M-AAT) gene, thus achieving concomitant PiZ-AAT knockdown in the liver and increased expression of M-AAT. [Mol Ther] Abstract

Suprachoroidal Electrotransfer: A Nonviral Gene Delivery Method to Transfect the Choroid and the Retina without Detaching the Retina
Investigators have developed a novel transfection method in the adult rat, called suprachoroidal electrotransfer, combining the administration of nonviral plasmid DNA into the suprachoroidal space with the application of an electrical field. [Mol Ther] Abstract

CEA Promoter-Regulated Oncolytic Adenovirus-Mediated Hsp70 Expression in Immune Gene Therapy for Pancreatic Cancer
In this paper, a carcinoembryonic antigen (CEA) promoter-regulated oncolytic adenovirus vector was constructed. The reporter gene assay demonstrated that the viral vector was confirmed to have tumor-specific replication features. [Cancer Lett] Abstract

Immunodominant, Liver-Specific Expression Suppresses Transgene-Directed Immune Responses in Murine Pompe Disease
An adeno-associated virus (AAV) vector containing a liver-specific regulatory cassette to drive acid α-glucosidase (GAA) expression (AAV-LSPhGAA) established immune tolerance in GAA knockout mice, whereas ubiquitous expression with AAV-CBhGAA provoked immune responses. Therefore, researchers investigated the hypothesis that immune tolerance induced by hepatic-restricted expression was dominant. [Hum Gene Ther] Abstract

BRCA1 Gene Therapy Reduces Systemic Inflammatory Response and Multiple Organ Failure and Improves Survival in Experimental Sepsis
Researchers hypothesized that gene therapy with BRCA1 would attenuate the sequelae of sepsis and peritonitis in mice subjected to caecal ligation and perforation and thioglycollate stimulation. [Gene Ther] Abstract

Evaluation of the Use of an Induced Pluripotent Stem Cell Sheet for the Construction of Tissue-Engineered Vascular Grafts
In this study, investigators evaluated the use of a sheet created from induced pluripotent stem cell-derived vascular cells as a potential source for the construction of a tissue-engineered vascular graft. [J Thorac Cardiovasc Surg] Abstract

Transplantation of Mesenchymal Stem Cells Promotes the Alternative Pathway of Macrophage Activation and Functional Recovery after Spinal Cord Injury
The present study was designed to investigate the effects of mesenchymal stem cell transplantation with a special focus on their effect on macrophage activation after spinal cord injury. [J Neurotrauma] Abstract

Human Umbilical Cord Mesenchymal Stromal Cells Mitigate Chemotherapy-Associated Tissue Injury in a Pre-Clinical Mouse Model
Researchers evaluated the prophylactic and therapeutic action of human umbilical cord mesenchymal stromal cells on the chemotherapy agent adriamycin-induced side-effects in two different tumor models. [Cytotherapy] Abstract

Enhance Progenitor Proliferation With Human LDL


BrainStorm Announces Clinical Data Supporting Safety and Efficacy of NurOwn™ Based on Initial Patient Results
BrainStorm Cell Therapeutics Inc. announced that the data from the initial patients in its amyotrophic lateral sclerosis Phase I/II human clinical trial treated with its NurOwn™ technology did not present any significant side effects and that the NurOwn™ treatment has so far proven to be safe. [BrainStorm Cell Therapeutics Inc.] Press Release

Celldex Therapeutics Announces Initiation of Phase I Clinical Trial of CDX-301
Celldex Therapeutics, Inc. announced that dosing has initiated in a Phase I study of its hematopoietic growth factor, CDX-301, in healthy subjects. CDX-301 is a potent stem cell mobilizer and dendritic cell growth factor. [Celldex Therapeutics, Inc.] Press Release

Tech Bulletin: Expansion Of Hematopoietic Stem And Progenitor Cells


Fleshing Out the US Alzheimer’s Strategy
In December 2010, the US Congress passed the National Alzheimer’s Project Act. The law instructs the US government to develop its first-ever strategic plan for battling Alzheimer’s disease, the dementia-inducing brain disorder that is expected to afflict at least 11 million US citizens by 2050. [United States Government, United States] Press Release

Charity Funding Boosts Controversial Therapy for Mitochondrial Disease
Wellcome Trust announced more than £4 million in support for a pioneering, and potentially controversial, IVF treatment that could prevent some forms of muscular dystrophy and other diseases caused by defective mitochondria. [Wellcome Trust, United Kingdom] Press Release

For Its New Science Minister Brazil Picks A Scientist
Brazil has announced that Marco Antônio Raupp, currently president of the Brazilian Space Agency, will become the new minister of science, technology and innovation on 24 January. [The Brazilian Government, Brazil]
Press Release


Alnylam to Restructure Organization, Aligning Resources with Focus on Highest Value Pipeline Opportunities in “Alnylam 5×15™” Product Strategy
Alnylam Pharmaceuticals, Inc. announced that it intends to implement a strategic corporate restructuring, including an approximate 33% reduction in its workforce, as it aligns its resources to focus on what it believes to be the company’s highest value opportunities with accelerated clinical development plans. [Alnylam Pharmaceuticals, Inc.] Press Release

Regenerative Medicine Company Chooses TAP Biosystems to Develop Custom Automation For Consistent Manufacturing of a Leading Cell-Based Therapy
TAP Biosystems announced it is collaborating with a major regenerative medicine company, to develop a custom manufacturing system to ensure the consistent quality of its innovative cell-based therapy. [TAP Biosystems] Press Release

Entrepreneurial Physician-Scientist to Lead New Center for Regenerative Medicine at Oregon Health & Science University
Kenton Gregory, M.D. is directing a new Center for Regenerative Medicine at Oregon Health & Science University. [Oregon Health & Science University] Press Release

Penn Lung Biologists to Receive $2.5 Million to Study Repair and Regeneration
The Perelman School of Medicine at the University of Pennsylvania (Penn) is one of six institutions to be named part of the National Heart, Lung, and Blood Institute’s Lung Repair and Regeneration Consortium. Each of the institutions will receive $2.5 million over five years. [Perelman School of Medicine, University of Pennsylvania] Press Release

Leading Eye Institute to Participate in ACT’s Embryonic Stem Cell Clinical Trial for Macular Degeneration
Advanced Cell Technology, Inc. (ACT) announced that the Wills Eye Institute in Philadelphia has received institutional review board approval as a site for the Phase I/II clinical trial for dry age-related macular degeneration using human embryonic stem cell-derived retinal pigment epithelial cells. [Advanced Cell Technology, Inc.] Press Release

Making Safer Chemicals: New NIH Grant Helps Researchers Study How Chemicals in Drugs and Around Us Impact Stem Cells
Bioengineers at Rensselaer Polytechnic Institute and the University of California, Berkeley, have been awarded a more than $2 million grant from the National Institutes of Health (NIH) to study how chemicals in drugs and our environment impact our stem cells. [Rensselaer Polytechnic Institute] Press Release

Academic Institutions Won Most of the California Institute for Regenerative Medicine’s $1.3 Billion in Awards over Past Five Years
Focus of California’s stem cell agency has been on setting up a foundation for leadership. [Genetic Engineering & Biotechnology News] Press Release

CIRM Allocates $40 Million to Support Stem Cell Genomics, Speeding Development of Therapies for Patients
California stem cell scientists will have a new resource for advancing therapies with a $40 million initiative approved by the governing board of the California Institute for Regenerative Medicine (CIRM). [California Institute for Regenerative Medicine] Press Release

Sernova Receives Patent Allowance in Japan
Sernova Corp. announced that the Japan Patent Office has issued a Notice of Allowance to Sernova for a patent entitled “Compositions Containing Sertoli Cells and Myoid Cells and Use Thereof in Cellular Transplants.” [Sernova Corp.] Press Release

PBL Awarded Pioneering US Patent on RNA Interference
Plant Bioscience Limited (PBL) is pleased to announce that the United States Patent and Trademark Office has issued out US Patent No. 8,097,710 with fundamental claims directed to methods of inducing gene silencing using short RNA molecules, or DNA constructs encoding short RNA molecules, in a wide range of organisms, including in plants and humans. [Plant Bioscience Limited] Press Release

St. Baldrick’s Foundation Gives Consortium $998,132 Grant for Childhood Cancer Research
The Childhood and Adolescent Lymphoma Cell Therapy Consortium, of which Baylor College of Medicine is a primary site, received a $998,132 grant from the St. Baldrick’s Foundation. [Baylor College of Medicine]
Press Release


Researchers Protest Against Minimum Cage Sizes for Breeding Rodents
US researchers are concerned that revised guidelines that recommend a minimum size for breeding lab rodents’ cages will substantially increase the cost of animal work. Press Release

Notice of Legislative Mandates in Effect for FY2012 (NOT-OD-12-034)

Notice of Salary Limitation on Grants, Cooperative Agreements, and Contracts (NOT-OD-12-035)

NIH Fiscal Policy for Grant Awards – FY 2012 (NOT-OD-12-036)

Notice of Revised NIMH Policies for Applications Submitted to PA-11-195 -“Midcareer Investigator Award in Patient-Oriented Research” (Parent K24) and PA-11-191 – “Independent Scientist Award” (Parent K02) (NOT-MH-12-013)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-953)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-1249)

National Cancer Institute; Notice of Meeting (FR Doc No: 2012-869)


February 10, 2012: Cellular, Tissue and Gene Therapies Advisory Committee Meeting: Waivers for Conflicts of Interest

Guidance for Industry: Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage


NEW Gordon Research Conference: Reprogramming Cell Fate
February 26-March 2, 2012
Galveston, United States

NEW 18th Annual Meeting of the German Society for Gene Therapy (DG-GT)
March 15-17, 2012
Frankfurt, Germany

NEW American Society of Gene & Cell Therapy 15th Annual Meeting and Course
March 16-19, 2012
Philadelphia, United States

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


Research and Development Technologist, hPSC Media (STEMCELL Technologies)

Scientist – Pluripotent Stem Cells (STEMCELL Technologies)

Research Technologist, Manufacturing Sciences (STEMCELL Technologies)

Business Analyst – Product Management (STEMSOFT Software)

Postdoctoral Fellow – Adult Neurogenesis and Neural Signaling (University of Kentucky)

Postdoctoral Position (The Laboratory for Equine and Comparative Orthopedic Research)

Research Associate II/III (Louisiana State University)

Associate Professor, Biology and Medicine in Maternal, Perinatal and Child Health (Wayne State University)

Senior Development Associate (Genzyme Corporation)

Scientist II, Innovation – Cell Processing Technologies (Lonza Walkersville, Inc.)

Senior Manager, Innovation – Cell Processing Technologies (Lonza Walkersville, Inc.)

Postdoctoral/Research Associate (Children’s Memorial Research Center)

Postdoctoral Fellow – Program in Children’s Regenerative Medicine (The University of Texas Medical School at Houston)

Postdoctoral Fellow and Technician Positions (North Carolina Central University)

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