Volume 13.00 | Jan 9

Issue 13.00 January 9, 2012
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Generation of Chimeric Rhesus Monkeys
Here, researchers demonstrate that rhesus monkey embryonic stem cells (ESCs) and isolated inner cell masses fail to incorporate into host embryos and develop into chimeras. However, chimeric offspring were produced following aggregation of totipotent cells of the four-cell embryos. [Cell] Abstract | Press Release

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PUBLICATIONS (Ranked by Impact Factor of the Journal)

Adult Human RPE Can Be Activated into a Multipotent Stem Cell that Produces Mesenchymal Derivatives
This study establishes the retinal pigment epithelial (RPE) stem cell as an accessible, human CNS-derived multipotent stem cell, useful for the study of fate choice, replacement therapy, and disease modeling. [Cell Stem Cell] Abstract | Press Release

Rejuvenation of Regeneration in the Aging Central Nervous System
Here researchers show that remyelination of experimentally induced demyelination is enhanced in old mice exposed to a youthful systemic milieu through heterochronic parabiosis. [Cell Stem Cell]
Abstract | Press Release

Somatic Progenitor Cell Vulnerability to Mitochondrial DNA Mutagenesis Underlies Progeroid Phenotypes in Polg Mutator Mice
Researchers found that Polg-Mutator mice had neural (NSC) and hematopoietic progenitor (HPC) dysfunction already from embryogenesis. N-acetyl-L-cysteine treatment rescued both NSC and HPC abnormalities, suggesting that subtle ROS/redox changes, induced by mtDNA mutagenesis, modulate somatic stem cell function. [Cell Metab] Abstract | Press Release

Essential Features and Rational Design of CRISPR RNAs that Function with the Cas RAMP Module Complex to Cleave RNAs
Researchers show that CRISPR-associated (Cas) RAMP module CRISPR RNAs (crRNAs) contain an 8 nucleotide 5 sequence tag (also found on crRNAs associated with other CRISPR-Cas pathways) that is critical for crRNA function and can be used to engineer crRNAs that direct cleavage of novel targets. [Mol Cell]
Abstract | Press Release

Implantation Site and Lesion Topology Determine Efficacy of a Human Neural Stem Cell Line in a Rat Model of Chronic Stroke
Magnetic resonance imaging here confirmed stroke damage and allowed the targeted injection of 450,000 human neural stem cells into peri-infarct tissue, rather than the lesion cyst. [Stem Cells] Abstract | Press Release

Foamy Virus Vector-Mediated Gene Correction of a Mouse Model of Wiskott–Aldrich Syndrome
In this study, researchers assessed the use of foamy virus vectors as a gene transfer system for Wiskott–Aldrich syndrome (WAS), using a Was knockout mouse model. [Mol Ther] Abstract

Concomitant Intravenous Nitroglycerin with Intracoronary Delivery of AAV1.SERCA2a Enhances Gene Transfer in Porcine Hearts
In an effort to enhance the efficiency and homogeneity of gene uptake in cardiac tissue, investigators examined the effects of nitroglycerin in a porcine model following AAV1.SERCA2a gene delivery. [Mol Ther] Abstract

Combined Tbet and IL12 Gene Therapy Elicits and Recruits Superior Antitumor Immunity In Vivo
This study was undertaken to determine whether gene therapy using combined Tbet and interleukin (IL)-12 complementary DNA would yield improved antitumor efficacy based on the complementary/synergistic action of these biologic modifiers. [Mol Ther] Abstract

Discovering Small Molecules that Promote Cardiomyocyte Generation by Modulating Wnt Signaling
Researchers have developed a robust in vivo small-molecule screen that modulates heart size and cardiomyocyte generation in zebrafish. Increased cardiomyocyte number in Cardionogen-treated embryos was due to expansion of cardiac progenitor cells. [Chem Biol] Abstract

Targeted Lentiviral Vectors Pseudotyped with the Tupaia paramyxovirus Glycoproteins
Researchers demonstrate here that lentiviral vectors can also be pseudotyped with the glycoproteins of Tupaia paramyxovirus, the hemagglutinin and fusion protein. [Gene Ther] Abstract

The Effects of Mechanical Stress on the Growth, Differentiation, and Paracrine Factor Production of Cardiac Stem Cells
In this study, human cardiac stem cells were seeded in a silicon chamber and mechanical stress was then induced by cyclic stretch stimulation (60 cycles/min with 120% elongation). [PLoS One] Abstract

Muscle-Derived Stem/Progenitor Cell Dysfunction Limits Healthspan and Lifespan in a Murine Progeria Model
Here researchers show that muscle-derived stem/progenitor cells (MDSPCs) from old and progeroid mice are defective in proliferation and multilineage differentiation. Intraperitoneal administration of MDSPCs, isolated from young wild-type mice, to progeroid mice confer significant lifespan and healthspan extension. [Nat Commun] Abstract | Press Release

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Alnylam Reports Positive Preliminary Clinical Results for ALN-PCS, an RNAi Therapeutic Targeting PCSK9 for the Treatment of Severe Hypercholesterolemia
ALN-PCS demonstrated statistically significant RNAi silencing of PCSK9 of up to 66% and reductions of up to over 50% in levels of low-density lipoprotein cholesterol, a clinically validated endpoint. [Alnylam Pharmaceuticals, Inc.] Press Release

Quark Pharmaceuticals Reports Favorable Interim Results from Phase I Clinical Study of QPI-1007
Quark Pharmaceuticals, Inc. announced interim results from the first two cohorts of its open label, first-in-human Phase I clinical study of QPI-1007, the Company’s proprietary synthetic siRNA drug candidate for ocular neuroprotection, in patients with recent onset of non-arteritic anterior ischemic optic neuropathy. [Quark Pharmaceuticals, Inc.] Press Release

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National Institute of General Medical Sciences Reorganizes
The National Institute of General Medical Sciences (NIGMS) has established two new divisions. Each will administer existing NIGMS programs along with programs transferred to NIGMS from the former National Institutes of Health National Center for Research Resources. [National Institutes of Health, United States]
Press Release

Singh Promises Windfall for India’s Scientists
India’s Prime Minister Manmohan Singh has vowed to more than double his nation’s spending on R&D over the next 5 years and build two major research facilities. [India’s Prime Minister, India] Press Release

Spain Cuts Science Ministry in Government Changeover
Spain’s ministry of science is the latest casualty of changes to the country’s government. Mariano Rajoy, leader of the newly elected right-wing People’s Party that took over, pledged to reduce the number of ministries from 15 to 12 to save money and improve governance. [Ministry of Science, Spain] Press Release

Privately-Funded Science University Plan
A new type of privately-funded science and technology university has been announced by the universities minister. [Universities Minister, United Kingdom] Press Release

Phacilitate Cell and Gene Therapy Forum 2012


Alnylam and Arrowhead Form Collaboration and Licensing Agreement
Alnylam Pharmaceuticals, Inc. and Arrowhead Research Corporation announced that they have entered into a collaboration and joint licensing agreement. [Alnylam Pharmaceuticals, Inc.] Press Release

Osiris Therapeutics Provides Update on Groundbreaking Stem Cell Trial for Type 1 Diabetes
Osiris Therapeutics, Inc. provided an update on its Phase II trial evaluating Prochymal, a formulation of adult mesenchymal stem cells, in patients with newly diagnosed type 1 diabetes. [Osiris Therapeutics, Inc.]
Press Release

Phase III Clinical Trial Begins for Prostate Cancer Immunotherapy
Advantagene Inc. and the University of Massachusetts Medical School have announced the beginning of a Phase III clinical study to test the ability of a novel immunotherapy to kill tumor cells and stimulate a patient-specific anti-tumor immune response for patients newly diagnosed with localized prostate cancer. [The University of Massachusetts Medical School] Press Release

ThermoGenesis Announces Approval of AXP® by India Ministry of Health
ThermoGenesis Corp. said that India’s Ministry of Health has approved the AutoXpress® System (AXP), enabling the Company to initiate commercial sales of the product in India. The AXP is used for the processing of stem cells from cord blood. [ThermoGenesis Corp.] Press Release

Neuralstem’s NSI-189 Trial in Major Depressive Disorder Receives FDA Approval to Advance to Phase Ib
Neuralstem, Inc. announced that it has been approved by the Food and Drug Administration (FDA) to advance to Phase Ib in its ongoing clinical trial to test its novel neuroregenerative compound, NSI-189, for the treatment of major depressive disorder. [Neuralstem, Inc.] Press Release

AlloCure’s Investigational Treatment for Acute Kidney Injury Receives Fast Track Designation by FDA
AlloCure, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AC607, the company’s investigational treatment for acute kidney injury. [AlloCure, Inc.] Press Release

Amsterdam Molecular Therapeutics Receives US Orphan Designation for Hemophilia B Gene Therapy
Amsterdam Molecular Therapeutics announced that the U.S. Food and Drug Administration has granted orphan drug designation to its gene therapy program for the treatment of hemophilia B. [Amsterdam Molecular Therapeutics] Press Release

Athersys Receives U.S. Patent Covering the Treatment of Cardiovascular Disease with Adult Stem Cells
Athersys, Inc. announced that it has been granted U.S. patent 8,075,881 that covers the use of non-embryonic multipotent stem cells for the treatment of cardiovascular conditions including myocardial infarction, or heart attack, congestive heart failure, myocardial ischemia and other heart conditions. [Athersys, Inc.] Press Release

Alnylam Files Clinical Trial Application for ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin for the Treatment of Transthyretin-Mediated Amyloidosis
Alnylam Pharmaceuticals, Inc. announced that it has filed a Clinical Trial Application with the U.K. Medicines and Healthcare products Regulatory Agency to initiate a Phase I clinical trial with ALN-TTR02, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis. [Alnylam Pharmaceuticals, Inc.] Press Release

Dicerna Pharmaceuticals Appoints Life Sciences Executive James B. Weissman to Chief Business Officer
Dicerna Pharmaceuticals, Inc. announced the appointment of James B. Weissman to chief business officer. [Dicerna Pharmaceuticals, Inc.] Press Release

Tengion Announces Relocation of Corporate Headquarters to Current Facility in Winston-Salem, North Carolina
Tengion, Inc. announced it has completed the relocation of its corporate headquarters from East Norriton, Pennsylvania to the Company’s existing facility in Winston-Salem, North Carolina. [Tengion, Inc.] Press Release

Major US Research Award for Professor Frank Barry
One of Ireland’s leading scientists working in the field of stem cell therapy is to receive the 2012 Marshall R. Urist Award for Excellence in Tissue Regeneration Research. [The National University of Ireland, Galway]
Press Release


A Present for NIH: President Signs Law Creating New Translational Center
A year after advisers made the proposal and following months of controversy, the National Institutes of Health (NIH) has secured a new translational research center. Press Release

Notice of NIH’s Creation of the National Center for Advancing Translational Sciences and Anticipated Implementation Plan (NOT-OD-12-027)

Notice of NIH Plans to Transfer All Programs, Awards, and Applications Associated with the National Center of Research Resources to Other NIH Institutes and Centers (NOT-OD-12-026)

Request for Information on Value of Information Research (NOT-HL-12-002)

Pediatric Phase 1/Pilot Consortium (Limited Competition UM1) (RFA-CA-12-502)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2011-33836)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2011-33833)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2011-33481)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2011-33492)


Consumer Information on Stem Cells

2012 Advisory Committee Tentative Meetings

Guidance for Industry: Current Good Tissue Practice and Additional Requirements for Manufacturers of Human Cells, Tissues, and Cellular and Tissue-Based Products

February 10, 2012: Cellular, Tissue and Gene Therapies Advisory Committee Meeting Announcement


Food and Drug Administration (United States)

FDA Warns About Stem Cell Claims
Food and Drug Administration (FDA) cautions consumers to make sure that any stem cell treatment they are considering has been approved by FDA or is being studied under a clinical investigation that has been submitted to and allowed to proceed by FDA. Press Release

Amendments to Regulations on Citizen Petitions, Petitions for Stay of Action, and Submission of Documents to Dockets (Docket No. FDA-2011-N-0697)

Guidance for Industry: Current Good Tissue Practice and Additional Requirements for Manufacturers of Human Cells, Tissues, and Cellular and Tissue-Based Products; Availability (Docket No. FDA-2008-D-0659)

Food and Drug Administration Transparency Initiative: Food and Drug Administration Report on Good Guidance Practices: Improving Efficiency and Transparency; Availability (Docket No. FDA-2009-N-0247)

Privacy Act of 1974; Report of an Altered System of Records, Including Addition of Routine Uses to an Existing System of Records; Bioresearch Monitoring Information System (Docket No. FDA-2011-N-0454)

Medicines and Healthcare Regulatory Agency (United Kingdom)

New MHRA Website Home Page
The Medicines and Healthcare Regulatory Agency (MHRA) is pleased to announce the launch of a brand new home page for their website which went live on Saturday 7 January 2012. Press Release

Therapeutic Goods Administration (Australia)

Resignation of Dr Rohan Hammett
The Parliamentary Secretary for Health, Catherine King, thanks the retiring National Manager of the Therapeutic Goods Administration, Dr Rohan Hammett. Press Release

Documents Released Under Section 11C of the Freedom of Information Act 1982

NEW Cell Culture World Congress 2012
February 28-March 1, 2012
Munich, Germany

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)

Postdoctoral Position – Center for Human Genetics (VIB Research Institute)

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)

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