ESC & iPSC News Volume 6.01 | Jan 12 2011

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Patient-Derived Embryonic Stem Cells Help Deliver “Good Genes” in a Model of Inherited Blood Disorder
Researchers report a gene therapy strategy that improves the condition of a mouse model of an inherited blood disorder, beta thalassemia. The gene correction involves using unfertilized eggs from afflicted mice to produce a batch of embryonic stem cell lines. [Press release from Nationwide Children’s Hospital discussing online prepublication in the Journal of Clinical Investigation]

Transforming Skin Cells Into Cartilage
Researchers at the Osaka University Graduate School of Medicine used fibroblasts isolated from adult mouse skin and expressed proteins used to induce pluripotency along with a factor that promotes a chondrocyte fate. The resulting cells resembled chondrocytes and produced cartilage when injected into mice. [Press release from EurekAlert! discussing online prepublication in the Journal of Clinical Investigation]

Embryonic Stem Cells Are a Potentially Unlimited Source of Functional Platelets for Transfusion
Advanced Cell Technology, Inc. reported that its hemangioblast-based technology can be used to generate functional platelets from human embryonic stem cells (hESCs). This research shows that it is feasible to generate functional megakaryocytes and platelets from hESCs on a large scale. [Press release from Stem Cell & Regenerative Medicine International, Inc. discussing online prepublication in Cell Research]

Calcineurin-NFAT Signaling Critically Regulates Early Lineage Specification in Mouse Embryonic Stem Cells and Embryos
Results demonstrate that the NFAT and Erk1/2 cascades form a signaling switch for early lineage segregation in mouse embryonic stem cells (ESCs) and provide significant insights into the regulation of the balance between ESC self-renewal and early lineage specification. [Cell Stem Cell]

A Human iPSC Model of Hutchinson Gilford Progeria Reveals Vascular Smooth Muscle and Mesenchymal Stem Cell Defects
To understand the underlying molecular pathology of Hutchinson-Gilford Progeria syndrome (HGPS), researchers derived induced pluripotent stem cells from HGPS dermal fibroblasts. [Cell Stem Cell]

Reprogramming Factor Expression Initiates Widespread Targeted Chromatin Remodeling
Data provide evidence for an early, organized, and population-wide epigenetic response to ectopic reprogramming factors that clarify the temporal order through which somatic identity is reset during reprogramming. [Cell Stem Cell]

Gene Therapy by Allele Selection in a Mouse Model of Beta-Thalassemia
Researchers present proof of principle that, for diseases associated with dominant alleles (gain-of-function or haploinsufficient loss-of-function), disease allele-free embryonic stem cells can be derived from afflicted individuals without genome manipulation. [J Clin Invest]

Generation of Hyaline Cartilaginous Tissue from Mouse Adult Dermal Fibroblast Culture by Defined Factors
Researchers show that retroviral expression of two reprogramming factors (c-Myc and Klf4) and one chondrogenic factor (SOX9) induces polygonal chondrogenic cells directly from adult dermal fibroblast cultures. [J Clin Invest]

Platelets Generated from Human Embryonic Stem Cells Are Functional In Vitro and in the Microcirculation of Living Mice
Researchers report that it is feasible to generate functional megakaryocytes and platelets from human embryonic stem cells on a large scale. [Cell Res]

Reprogramming of Ovine Adult Fibroblasts to Pluripotency via Drug-Inducible Expression of Defined Factors
Researchers report that sheep somatic cells can be directly reprogrammed to induced pluripotent stem (iPS) cells using defined factors (Oct4, Sox2, c-Myc, Klf4, Nanog, Lin28, SV40 large T and hTERT). Their observations indicated that somatic cells from sheep are more difficult to reprogram than somatic cells from other species, in which iPS cells have been reported. [Cell Res]

Genome-Wide Studies Reveal That Lin28 Enhances the Translation of Genes Important for Growth and Survival of Human Embryonic Stem Cells
Researchers suggest that Lin28, working in concert with RNA helicase A, enhances the translation of genes important for the growth and survival of human embryonic stem cells. [Stem Cells]

Combined Chemical Treatment Enables Oct4-Induced Reprogramming from Mouse Embryonic Fibroblasts
Researchers reported identification of a new small molecule, a protein arginine methyltransferase inhibitor AMI-5, which enabled Oct4-induced reprogramming of MEFs in combination with TGF-beta inhibitor A-83-01. [Stem Cells]

A Targeted NKX2.1 Hesc Reporter Line Enables Identification of Human Basal Forebrain Derivatives
The study highlights the diversity of cell types that can be generated from human NKX2.1⁺ progenitors and demonstrate the utility of NKX2.1^GFP/w human embryonic stem cells for investigating human forebrain development and neuronal differentiation. [Stem Cells]

INDUSTRY NEWS

Horizon and University of Washington Announce Collaboration to Apply rAAV Gene Editing in iPS cells
Horizon Discovery and the University of Washington announced that they have entered into a two-year research collaboration aimed at generating a process for gene editing in inducible human pluripotent stem cells. [Horizon Discovery Limited Press Release]

Cell Medica Signs an Exclusive License Agreement and Strengthens Collaboration with the Center for Cell and Gene Therapy, Baylor College of Medicine
Cell Medica announced an exclusive license agreement and research collaboration with the Center for Cell and Gene Therapy, Baylor College of Medicine (Houston, Texas), for the commercialization of an innovative cell-based treatment for cancers associated with the oncogenic Epstein Barr virus. [Cell Medica Limited Press Release]
 
VistaGen Therapeutics and NuPotential Receive NIH Grant to Develop Safer Approaches for Producing Patient-Specific Induced Pluripotent Stem Cells
VistaGen Therapeutics, Inc. and NuPotential, Inc. announced that the National Heart, Lung and Blood Institute of the U.S. National Institutes of Health (NIH) has awarded to the companies a grant of approximately $500,000 to accelerate development of novel and safer approaches to generate patient-specific induced pluripotent stem cells for regenerative medicine, drug discovery and drug rescue. [Business Wire]

The Power of “Convergence”
A new model for scientific research known as “convergence” offers the potential for revolutionary advances in biomedicine and other areas of science, according to a white paper issued today by 12 leading Massachusetts Institute of Technology researchers. [Massachusetts Institute of Technology Press Release]

How New COMPETES Science Law Broadens NSF Education Programs
Only one of the seven directorates at the National Science Foundation (NSF) has “education” in its title. But the reauthorization of the America COMPETES Act spells out program changes meant to ensure that every component of the $7 billion agency is involved in training the next generation of researchers and improving public scientific literacy. [National Science Foundation, United States]

Amid Legal Uncertainties, NIH Approves More Embryonic Stem Cells
The legal battle over whether taxpayer dollars can go toward human embryonic stem cells research continues to drag on, but the US National Institutes of Health (NIH) is not waiting for a final court decision before adding new cell lines to its list of those eligible for financial backing. [National Institutes of Health, United States]

Despite Dire Budget Outlook, Panel Tells NIH to Train More Scientists
Despite the grim employment picture for would-be academic researchers, the National Institutes of Health (NIH) should maintain or even increase the number of graduate students and postdocs it supports. [National Institutes of Health, United States]
 
FDA Launches Website to Help Regulated Industries Save Time, Resources
The U.S. Food and Drug Administration introduced a new web resource called FDA Basics for Industry (www.fda.gov/FDABasicsforIndustry) to help companies and others save time and resources in their interactions with the agency. [Food and Drug Administration, United States]

FDA's Number Two Official Leaving for Maryland
Joshua Sharfstein, the number two official at the U.S. Food and Drug Administration (FDA), is leaving next week to head health services for the State of Maryland, according to news reports. The 41-year-old pediatrician has been a key player with FDA Commissioner Margaret Hamburg in reforming the agency over the past 2 years. [Food and Drug Administration, United States]

Hamburg Names John Taylor as Acting FDA Deputy
U.S. Food and Drug Administration (FDA) commissioner Dr. Margaret Hamburg named her office's top lawyer, John Taylor III, to be the temporary replacement for outgoing deputy commissioner Dr. Joshua Sharfstein. [Food and Drug Administration, United States]

ESF Moves Toward Rebirth, But Change Worries Some
2011 will likely see the end of the European Science Foundation (ESF), at least in its current form. After 37 years of funding researchers, organizing collaborations, and convening meetings across the continent, ESF is expected to transform into a lobbying and strategy organization. [European Science Foundation, European Union]

A New Pathway for the Regulation and Governance of Health Research
The Academy's review, 'A new pathway for the regulation and governance of health research' was published. The report proposes four key principles that should underpin the regulation and governance framework around health research in the UK and makes recommendations. [The Academy of Medical Sciences, United Kingdom]

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-385) [National Institutes of Health, United States]

National Cancer Institute; Notice of Closed Meeting (FR Doc. 2011-253) [National Institutes of Health, United States]
 
Center for Scientific Review; Amended Notice of Meeting (FR Doc. 2011-255) [National Institutes of Health, United States]
 
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-257) [National Institutes of Health, United States]
 
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-256) [National Institutes of Health, United States]
 
FDA Transparency Initiative: Improving Transparency to Regulated Industry [Docket No. FDA-2009-N-0247] [Food and Drug Administration, United States]
 
Draft Guidance for Industry on Electronic Source Documentation in Clinical Investigations; Availability [Docket No. FDA-2010-D-0643] [Food and Drug Administration, United States]