Volume 13.03 | Jan 30

Issue 13.03 January 30, 2012
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Researchers Develop Gene Therapy that Could Correct a Common Form of Blindness
A new gene therapy method developed by University of Florida researchers has the potential to treat a common form of blindness that strikes both youngsters and adults. The technique works by replacing a malfunctioning gene in the eye with a normal working copy that supplies a protein necessary for light-sensitive cells in the eye to function. [Press release from the University of Florida discussing online prepublication in the Proceedings of the National Academy of Sciences, USA] Press Release | Abstract

Learn More - Reduce Costs Associated With Collecting And Culturing Cells By Using Frozen Primary Cells

PUBLICATIONS (Ranked by Impact Factor of the Journal)

Probing Sporadic and Familial Alzheimer’s Disease Using Induced Pluripotent Stem Cells
Here investigators reprogrammed primary fibroblasts from two patients with familial Alzheimer’s disease, both caused by a duplication of the amyloid-ß precursor protein gene, two with sporadic Alzheimer’s disease and two non-demented control individuals into induced pluripotent stem cell lines. [Nature] Abstract | Press Release

First Experience in Humans Using Adipose Tissue-Derived Regenerative Cells in the Treatment of Patients with ST-Segment Elevation Myocardial Infarction
The APOLLO trial is a randomized, double-blind, placebo-controlled, phase I/IIa study designed to assess the safety and feasibility of intracoronary infusion of adipose tissue-derived regenerative cells in the treatment of patients in the acute phase of a large ST-segment elevation acute myocardial infarction. [J Am Coll Cardiol] Abstract | Press Release

Recurrent Genomic Instability of Chromosome 1q in Neural Derivatives of Human Embryonic Stem Cells
Here, scientists report that neural differentiation of human embryonic stem cells (hESCs) systematically produced a neural stem cell population that could be propagated for more than 50 passages without entering senescence; this was true for all six hESC lines tested. [J Clin Invest] Abstract | Press Release

Adipose Tissue-Derived Stem Cell-Seeded Small Intestinal Submucosa for Tunica Albuginea Grafting and Reconstruction
The aim of this rat study was to assess the feasibility of seeding adipose tissue-derived stem cells onto small intestinal submucosa grafts for tunica albuginea reconstruction. [Proc Natl Acad Sci USA]
Abstract | Press Release

Simultaneous Gene Transduction and Silencing Using Stimuli-Responsive Viral/Nonviral Chimeric Nanoparticles
An adeno-associated viral particle was shielded with acid-degradable, siRNA-encapsulating polyketal shell, resulting in core-shell viral/nonviral chimeric nanoparticles. [Biomaterials] Abstract

Cell Fate Control Gene Therapy Based on Engineered Variants of Human Deoxycytidine Kinase
Investigators describe a novel cell fate control gene therapy system based on engineered variants of human deoxycytidine kinase that overcomes limitations of current modalities. [Mol Ther] Abstract

Mechanical Loading of Stem Cells for Improvement of Transplantation Outcome: The Role of Loading History
In this study researchers hypothesized that the mechanical loading history of muscle derived stem cells (MDSCs) would significantly impact MDSC survival, host tissue angiogenesis, and myocardial function following MDSC transplantation into acutely infarcted myocardium. [Tissue Eng Part A] Abstract

AAV-Based shRNA Silencing of NF-κB Ameliorates Muscle Pathologies in mdx Mice
In this study, researchers used the recombinant adeno-associated virus serotype 9 (AAV9) carrying a short hairpin RNA (shRNA) specifically targeting the messenger RNA of NF-kappa B (NF-κB)/p65 (p65-shRNA), the major subunit of NF-κB associated with chronic inflammation in mdx mice. [Gene Ther] Abstract

Systemic Delivery of scAAV9 in Fetal Macaques Facilitates Neuronal Transduction of the Central and Peripheral Nervous Systems
Because of the clinical relevance of the non-human primate (NHP) model, researchers investigated the ability of AAV9 to transduce the NHP CNS following intrauterine gene therapy. [Gene Ther] Abstract

Transplantation of Human Central Nervous System Stem Cells – Neuroprotection in Retinal Degeneration
In the present study, researchers explored the efficacy of human central nervous system stem cells in a rodent model of retinal degeneration and extended their previous cell transplantation studies to include an in-depth examination of donor cell behavior and phenotype post-transplantation. [Eur J Neurosci] Abstract

Tech Bulletin: Expansion Of Hematopoietic Stem And Progenitor Cells


Marina Biotech Starts Cohort 2 of Its Phase Ib START-FAP Clinical Trial of CEQ508
Marina Biotech, Inc. announced that Cohort 2 in the Dose Escalation Phase of the START-FAP (Safety and Tolerability of An RNAi Therapeutic in Familial Adenomatous Polyposis) clinical trial with CEQ508 will begin enrolling patients in the next several weeks at Massachusetts General Hospital in Boston. [Marina Biotech, Inc.] Press Release

Cytori Receives Approval from FDA to Initiate U.S. Cardiac Cell Therapy Trial; Investigational Device Exemption (IDE) Approved for ATHENA
Cytori Therapeutics has received an IDE approval from the U.S. Food and Drug Administration (FDA) to begin the ATHENA trial. ATHENA will investigate the use of the Celution® System, an innovative medical device to prepare adipose-derived stem and regenerative cells to treat a form of coronary heart disease, chronic myocardial ischemia. [Cytori Therapeutics, Inc.] Press Release

Watch The Video: Performing CFC Assays Using MethoCult™


Leading Global Cell Therapy Organizations Support U.S. Department of Justice Appeal of Ruling on Donor Compensation
A coalition of nine leading international health organizations issued a statement supporting the U.S. Department of Justice’s appeal of the Ninth Circuit Court ruling that allows certain marrow donors to be compensated. [U.S. Department of Justice, United States] Press Release

National Academy Picks UC Administrator for Top Staff Job
Bruce Darling, now vice president for laboratory management at the University of California (UC), was named to the job of overseeing day-to-day operations at U.S. National Academy of Sciences and its operating arm, the National Research Council. [U.S. National Academy of Sciences, United States] Press Release

Research in Asia Heats Up
US indicators reveal challenges and opportunities as science momentum shifts to China. [US National Science Board, United States] Press Release

United Kingdom Sets Sights on Gene Therapy in Eggs
Public consultation and safety assessment would pave the way for embryo manipulation to treat genetic diseases. [Human Fertilisation and Embryology Authority, United Kingdom] Press Release


Creighton to Explore Usage of Adult Stem Cells to Improve Angioplasty Results
A Creighton University School of Medicine researcher has received a $3.3 million, five-year grant from the National Institutes of Health to study what role adult stem cells might play in repairing damaged coronary arteries. [Creighton University] Press Release

BioTime Licenses Technology for Key Regulatory Gene Underlying Cancer and Stem Cell Reprogramming from The Wistar Institute
BioTime, Inc. announced that it has obtained an exclusive license from The Wistar Institute in Philadelphia, PA for technology related to a gene designated as SP100. Wistar Institute researchers have demonstrated pivotal roles for this gene in both cancer and stem cell biology. [BioTime, Inc.] Press Release

Amorcyte, a NeoStem Company, Enrolls First Patient in PreSERVE Phase II Trial for Acute Myocardial Infarction
Amorcyte, LLC, a NeoStem, Inc. company announced the enrollment of the first patient in the Amorcyte PreSERVE Phase II trial for acute myocardial infarction. The study is a multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of infarct-related artery infusion of AMR-001, an autologous bone marrow derived cell therapy enriched for CD34+ cells. [NeoStem, Inc.] Press Release

Vet-Stem Announces Milestone of 8,000 Animals Treated with Vet-Stem Cell Therapy
Vet-Stem Inc., announced that over 8,000 animals have now been treated with Vet-Stem cell therapy. [Vet-Stem Inc.] Press Release

Charter Medical Receives CE Mark Clearance for Next Generation Cell Freeze® Cryogenic Stem Cell Storage Containers
Charter Medical, Ltd., a Lydall subsidiary, announced that it received CE Mark approval on its next generation Cell Freeze® Cryogenic Storage Containers for Hematopoietic Progenitor Cells. [Lydall, Inc.] Press Release

World-Renowned Cell-Therapy Researcher, Doris Taylor, PhD, Joins Texas Heart Institute at St. Luke’s Episcopal Hospital
Officials at the Texas Heart Institute (THI) at St. Luke’s Episcopal Hospital announced that Doris Taylor, PhD, FAHA, FACC, one of the world’s leading cell therapy and cardiac regeneration scientists, will join THI beginning March 1, 2012. [Texas Heart Institute] Press Release

TAP Biosystems’ Project to Manufacture 3D Tissue Equivalents – Develops Potential Cell Therapy for Corneal Surface Disease
TAP Biosystems is pleased to announce that its Technology Strategy Board funded project with UCL scientists has made a significant advance towards developing a tissue-engineered, biomimetic cornea as a therapy. [TAP Biosystems] Press Release

Can Gene Therapy Be Used to Prevent Vein Graft Disease?
A grant of over £100,000 to investigate gene therapy in vein grafts that are used in heart bypass surgery has been awarded to Gavin Murphy, Reader in Cardiac Surgery in the School of Clinical Sciences at the University of Bristol from national heart charity, Heart Research UK. [University of Bristol] Press Release

French Institute Prepares for Gene-Therapy Push
Scientific director Fulvio Mavilio has a mandate to sharpen the research profile of Genethon. One of his main strategies is to create international clinical networks for gene therapy around Genethon. [NatureNews]
Press Release

Alnylam Receives Notice of Allowance from United States Patent and Trademark Office (USPTO) for New Patent Broadly Covering Second Generation Lipid Nanoparticles (LNP) for Systemic Delivery of RNAi Therapeutics
Alnylam Pharmaceuticals, Inc. announced that the USPTO has issued a Notice of Allowance for patent application number 12/813,448, covering the company’s proprietary second generation LNP platform, including the “MC3” lipid. [Alnylam Pharmaceuticals, Inc.] Press Release

Prosensa Raises €23 Million in New Equity Financing Led by New Enterprise Associates
The fundraising will enable Prosensa to advance its portfolio of RNA-modulating therapeutics for the treatment of rare diseases, including Duchenne muscular dystrophy, Myotonic Dystrophy and Huntington’s disease. [Prosensa] Press Release

World-Class Scientists Chosen for HHMI’s First International Early Career Award
Top biomedical scientists from 12 countries will receive an important boost at a critical time in their careers from the Howard Hughes Medical Institute’s (HHMI’s) inaugural International Early Career Scientist awards. [Howard Hughes Medical Institute] Press Release


Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-1381)

Center for Scientific Review; Notice of Closed Meeting (FR Doc No: 2012-1699)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc No: 2012-1371)

Prospective Grant of Exclusive License: Development of T Cell Receptors and Chimeric Antigen Receptors into Therapeutics for Adoptive Transfer in Humans To Treat Cancer (FR Doc No: 2012-1383)

February 10, 2012: Cellular, Tissue and Gene Therapies Meeting: Agenda


Food and Drug Administration (United States)

Vaccines and Related Biological Products Advisory Committee; Notice of Meeting (FR Doc No: 2012-1456)


NEW The Jackson Laboratory: The Reproductive Biology of Breeding Mice
February 9, 12:00 PM (ET in US)

NEW Molecular Medicine Tri-Conference 2012
February 19-23, 2012
San Francisco, United States

NEW World Immune Regulation Meeting – VI
March 18-21, 2012
Davos, Switzerland

NEW American Society for Neural Therapy and Repair Annual Meeting
April 26-28, 2012
Clearwater Beach, 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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