Volume 14.09 | Mar 11

Issue 14.09 March 11, 2013
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Publications | Reviews | Science | Policy | Business | NIH | CBER | Regulatory | Events | Jobs
TOP STORY
Researchers Utilize Genetically Corrected Stem Cells to Spark Muscle Regeneration
Researchers have combined genetic repair with cellular reprogramming to generate stem cells capable of muscle regeneration in a mouse model for duchenne muscular dystrophy. [Press release from The University of Minnesota discussing online prepublication in Nature Communications] Press Release | Abstract

New: Directed Differentiation of Pluripotent Stem Cells. A Cell Stem Cell Poster

 
PUBLICATIONS (Ranked by impact factor of the journal)

Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression
Researchers developed a method for controlling gene expression based on Cas9, an RNA-guided DNA endonuclease from a type II CRISPR system. [Cell] Abstract | Graphical Abstract | Press Release

Forebrain Engraftment by Human Glial Progenitor Cells Enhances Synaptic Plasticity and Learning in Adult Mice
To assess the cell-autonomous and species-selective properties of human glia, researchers engrafted human glial progenitor cells (GPCs) into neonatal immunodeficient mice. Upon maturation, the recipient brains exhibited large numbers and high proportions of both human glial progenitors and astrocytes. [Cell Stem Cell]
Abstract | Press Release | Video

Inhibition of Myc Family Proteins Eradicates KRas-Driven Lung Cancer in Mice
Investigators previously showed in a preclinical mouse model of non-small-cell lung cancer that systemic Myc inhibition using the dominant-negative Myc mutant Omomyc exerts a dramatic therapeutic impact, triggering rapid regression of tumors with only mild and fully reversible side effects. Using protracted episodic expression of Omomyc, they demonstrated that metronomic Myc inhibition not only contains Ras-driven lung tumors indefinitely, but also leads to their progressive eradication. [Genes Dev] Abstract | Press Release

Rare Somatic Cells from Human Breast Tissue Exhibit Extensive Lineage Plasticity
Scientists identified cell surface markers associated with repression of p16INK4a/cyclin-dependent kinase inhibitor 2A, a critical determinant in the acquisition of a plastic state. These cell surface markers allowed direct isolation of rare cells from healthy human breast tissue that exhibit extensive lineage plasticity. In vitro, in vivo, and teratoma assays demonstrated that either a directly sorted (uncultured) or a single-cell (clonogenic) cell population from primary tissue can differentiate into functional derivatives of each germ layer, ectodermal, endodermal, and mesodermal. [Proc Natl Acad Sci USA] Abstract | Full Article | Press Release

Ocular Complications in Children within 1 Year after Hematopoietic Stem Cell Transplantation
Investigators studied the development of ocular complications in children within one year after hematopoietic stem cell transplantation. [JAMA Ophthalmol] Abstract | Press Release

Adult Human Gingival Epithelial Cells as a Source for Whole-Tooth Bioengineering
Researchers showed that cells can be isolated from adult human gingival tissue that can be expanded in vitro and, when combined with mouse embryonic tooth mesenchyme cells, form teeth. Teeth with developing roots can be produced from this cell combination following transplantation into renal capsules. These bioengineered teeth contain dentin and enamel with ameloblast-like cells and rests of Malassez of human origin. [J Dent Res] Abstract | Press Release

Periurethral Skeletal Myofiber Implantation in Patients with Urinary Incontinence and Intrinsic Sphincter Deficiency: A Phase I Clinical Trial
In a phase I clinical trial, researchers investigated whether periurethral myofiber implantation generated local myogenesis and improved continence in ten patients (five men and five women) with intrinsic sphincter deficiency (ISD). They found that myofiber implantation increased intraurethral pressure and periurethral electromyographic activity in patients with ISD. There were no serious side-effects. [BJU Int] Abstract

Enhanced Tumor Suppression by Adenoviral PTEN Gene Therapy Combined with Cisplatin Chemotherapy in Small-Cell Lung Cancer
Investigators examined the combined antitumor effect of adenovirus-mediated phosphatase and tensin homolog deleted on chromosome 10 (PTEN) gene therapy and cisplatin chemotherapy on PTEN-null NCI-H446 human small-cell lung cancer cells in vitro and in vivo in athymic BALB/c nude mice. [Cancer Gene Ther] Abstract

New TeSR™-E8™ is Here For Feeder-Free Culture of Human ES Cells and iPS Cells



REVIEWS
From Bench to Bedside: Review of Gene and Cell-Based Therapies and the Slow Advancement into Phase III Clinical Trials, with a Focus on Aastrom’s Ixmyelocel-T
This review article provides an overview of the progression of research from gene therapy to the cellular therapy field as it applies to peripheral arterial disease, as well as the position of Aastrom’s cellular therapy, ixmyelocel-T, within this field. [Stem Cell Rev] Full Article

Learn more about the new smartphone app for human blood cell frequencies


SCIENCE NEWS

Creating New Tissue Instead of Transplanting Hearts
At the Vienna University of Technology, chemical substances have been developed, which turn the body’s own progenitor cells into functioning, beating heart cells. This discovery could open the door to a new kind of regenerative medicine. [Vienna University of Technology] Press Release | Video

Devices Aim to Deliver on Stem-Cell Therapies
Researchers designed a needle that bends. First, a straight, thin tube is injected into the brain and a flexible nylon catheter pushed through it. A deflector inside the tube arcs the catheter up and away from the entry track, and an even narrower plunger ejects cells from the catheter. In one injection, the device can deposit cells anywhere within a 2-centimeter radius along the track, a volume bigger than an entire mouse brain. [Nature News] Press Release

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Cell Therapy News is giving away a free registration for the 11th Annual International Cord Blood Symposium in San Francisco! A Forum for Education, Science and Medical Professionals. Click here to enter.


 
POLICY

Cora Marrett to Be Acting NSF Director
Cora Marrett will become acting director of the National Science Foundation (NSF) when Subra Suresh steps down. And while the temporary elevation of the veteran NSF administrator, now deputy director, comes as no surprise, some former directors are urging the White House to think seriously of giving her the job for real. [National Science Foundation, United States] Press Release

Senate Spending Bill Would Help NSF Preserve Its Priorities
Senate Democrats are hoping to make it easier for the National Science Foundation (NSF) and a handful of other federal science agencies to manage the impact of sequestration. The Senate’s approach would still result in a 5% cut to each agency’s budget. But NSF officials say it should give sufficient flexibility to honor funding commitments to current grantees, avoid furloughs, protect programs for young scientists, and continue work on large new facilities. [National Science Foundation, United States] Press Release



BUSINESS

Cellectis and Stemgent Partner to Provide Custom Genome-Engineered Induced Pluripotent Stem Cells
Cellectis bioresearch announced a collaboration agreement with Stemgent, Inc. to provide research services that combine mRNA reprogramming technology and genome engineering. The partnership marries Cellectis bioresearch’s leadership in genome engineering with Stemgent’s expertise in cellular reprogramming. [Cellectis bioresearch] Press Release

RXi Pharmaceuticals and OPKO Health Announce Strategic Pooling of RNAi Assets
RXi Pharmaceuticals Corporation and OPKO Health, Inc. announced a strategic pooling of their respective assets in the field of RNA interference (RNAi). According to the terms of an asset purchase agreement jointly executed by the parties, RXi will acquire substantially all of OPKO’s RNAi-related assets, including an extensive intellectual property portfolio. [RXi Pharmaceuticals Corporation] Press Release

ReNeuron Wins Two Major Biomedical Catalyst Grants to Pursue Core Stem Cell Therapy Programs
ReNeuron Group plc announced that it has been awarded two separate grants, totaling £1.2 million, from the UK Biomedical Catalyst to pursue further development of two of its core stem cell therapy candidates. The first award to ReNeuron of £0.4 million is a Late Stage Biomedical Catalyst grant and relates to the Company’s ReN009 stem cell therapy candidate for critical limb ischemia. The second award of £0.8 million is an Early Stage Biomedical Catalyst grant and relates to the Company’s ReN003 stem cell candidate for the treatment of retinitis pigmentosa. [ReNeuron Group plc] Press Release

NeoStem Reports Second Data Safety Monitoring Board Review: Continue with PreSERVE AMI Phase II Trial as Planned
NeoStem, Inc. announced that it received approval to continue the PreSERVE AMI Phase II clinical trial following its second interim data and safety review by the Data Safety Monitoring Board. The PreSERVE trial of AMR-001 is a Phase II, randomized, placebo controlled, double-blind study designed to treat 160 patients and is approved by the FDA to enroll up to 180 patients. [NeoStem, Inc.] Press Release

ImmunoCellular Therapeutics Announces Issuance of Key Patent for ICT-121 Cancer Vaccine
ImmunoCellular Therapeutics, Ltd. announced that the US Patent and Trademark Office has issued a key patent application covering ICT-121, an immunotherapeutic vaccine targeting CD133, an important cancer stem cell marker that is commonly overexpressed on a broad range of solid tumors. The patent includes claims covering composition of matter as well as methods of use. [Business Wire] Press Release

DCVax®-Direct Phase I/II Trial for All Inoperable Solid Tumors Is Expected to Produce Ongoing Results in 2013
Northwest Biotherapeutics announced that its Phase I/II DCVax-Direct clinical trial for all inoperable solid tumor cancers is planned to begin within approximately the next sixty days. [Northwest Biotherapeutics, Inc.]
Press Release

NW Bio Provides Guidance on Phase III Trial Enrollment Timing: Completion Expected to Be Faster or More Efficient than Relevant Comparison Trials
Northwest Biotherapeutics (NW Bio) announced that it expects to complete enrollment in its 312-patient Phase III clinical trial for glioblastoma multiforme brain cancer within a period that is faster or more efficient than relevant comparison trials with immune therapies for the same brain cancer. [Northwest Biotherapeutics, Inc.]
Press Release

Silence Therapeutics Gets German Approval for Cancer Drug Trial
Biotech research and development company Silence Therapeutics said it received approval from German authorities for trials of its pancreatic cancer drug and was reviewing financing options for an aggressive expansion. [Reuters] Press Release

Minerva Biotechnologies Announces Major Breakthrough in Human Stem Cell Research
Minerva Biotechnologies announced a major breakthrough in human stem cell research. Minerva scientists converted established human stem cells to the elusive “naïve” state and maintained them there indefinitely simply by culturing the cells in the dimeric form of a natural, but newly discovered human growth factor, called NM23-H1. [Business Wire] Press Release

UI Researchers Developing 3D Printer, ‘Bio-Ink’ to Create Human Organs
Using its facilities for engineering living tissue systems, the Biomanufacturing Laboratory at University of Iowa (UI) College of Engineering’s Center for Computer Aided Design is working to develop and refine various 3D printing processes required for organ and tissue fabrication. [The University of Iowa] Press Release



NIH

Bioengineering Research Grants (BRG)(R01)(PAR-13-137)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-04960)

National Institute on Drug Abuse; Notice of Closed Meetings (FR Doc No: 2013-05102)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-05365)



REGULATORY

Therapeutic Goods Administration (Australia)

Advisory Committee on Biologicals (ACB)



EVENTS

NEW The Jackson Laboratory: All You Need Is Love – Reproductive Biology of Mice
March 21, 1:00PM (ET in US)
Webinar

NEW 11th Annual International Cord Blood Symposium
June 6-8, 2013
San Francisco, United States

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



JOB OPPORTUNITIES

Faculty Position – Stem Cell, Vascular and Developmental Biology in Ophthalmology (Zhongshan Ophthalmic Center, Sun Yat-Sen University)

Postdoctoral Researcher (The University of Texas Health Science Center at San Antonio)

Postdoctoral Fellow – Transcriptional Regulation in CD8 T Lymphocytes (Université Libre de Bruxelles – Institute for Medical Immunology)

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

Postdoctoral Fellow – Pluripotent Stem Cells (Faculty of Medicine, University of Ottawa)

Lecturer or Reader – Associate Professor (University of Reading)

Cord Blood Laboratory Manager (Puget Sound Blood Center)


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