Volume 12.41 | Oct 17

Issue 12.41 October 17, 2011
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TOP STORY
Clean Correction of a Patient’s Genetic Mutation
For the first time, scientists have cleanly corrected a human gene mutation in a patient’s stem cells. [Press release from Wellcome Trust Sanger Institute discussing online prepublication in Nature] Press Release | Abstract

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PUBLICATIONS (Ranked by Impact Factor of the Journal)
Correction of Sickle Cell Disease in Adult Mice by Interference with Fetal Hemoglobin Silencing
Here, scientists show that the repressor BCL11A is required in vivo for silencing of γ-globin expression in adult animals, yet dispensable for red cell production. [Science] Abstract | Press Release

Therapeutic siRNA Silencing in Inflammatory Monocytes in Mice
Here researchers present an optimized lipid nanoparticle and a CCR2-silencing short interfering RNA (siRNA) that, when administered systemically in mice, show rapid blood clearance, accumulate in spleen and bone marrow, and localize to monocytes. [Nat Biotechnol] Abstract | Press Release

CD140a Identifies a Population of Highly Myelinogenic, Migration-Competent and Efficiently Engrafting Human Oligodendrocyte Progenitor Cells
To establish a more efficient means of isolating myelinogenic oligodendrocyte progenitor cells (OPCs), investigators sorted fetal human forebrain cells for CD140a, an epitope of platelet derived growth factor receptor α, which is differentially expressed by OPCs. [Nat Biotechnol] Abstract | Press Release

Effect of Donor-Recipient HLA Matching at HLA A, B, C, and DRB1 on Outcomes After Umbilical-Cord Blood Transplantation for Leukemia and Myelodysplastic Syndrome: A Retrospective Analysis
Researchers used Cox regression to assess retrospectively the effect of donor-recipient HLA matching on outcomes of single umbilical-cord blood transplantations for leukemia and myelodysplastic syndrome. [Lancet Oncol] Abstract | Press Release

Parabronchial Smooth Muscle Constitutes an Airway Epithelial Stem Cell Niche in the Mouse Lung After Injury
Here, investigators report that the Wnt/fibroblast growth factor 10 embryonic signaling cascade is reactivated in mature parabronchial SMCs after naphthalene-induced injury to airway epithelium. [J Clin Invest] Abstract | Press Release

Blimp1 Expression Predicts Embryonic Stem Cell Development In Vitro
Here researchers show that inner cell mass-generated cells expressing Blimp1, a key transcriptional repressor of the somatic program during germ cell specification, emerge on day 2 of blastocyst culture. [Curr Biol] Abstract | Press Release

Rapid and Efficient Reprogramming of Somatic Cells to Induced Pluripotent Stem Cells By Retinoic Acid Receptor Gamma and Liver Receptor Homolog 1
Here scientists report that enhancing retinoic acid (RA) signaling by expressing RA receptors (RARs) or by RA agonists profoundly promoted reprogramming, but inhibiting it using a RAR-α dominant-negative form completely blocked it. [Proc Natl Acad Sci USA] Abstract | Press Release

Why Is Less Cationic Lipid Required to Prepare Lipoplexes from Plasmid DNA than Linear DNA in Gene Therapy?
Herein for the first time, researchers experimentally show that for different types of cationic lipids, plasmid DNA, in contrast to linear DNA, is compacted with a large amount of its counterions, thus yielding a lower effective negative charge. [J Am Chem Soc] Abstract | Press Release

Insulin Biosynthesis in Neuronal Progenitors Derived from Adult Hippocampus and the Olfactory Bulb
In the present study, investigators demonstrated that insulin is produced not only in the mammalian pancreas but also in adult neuronal cells derived from the hippocampus and olfactory bulb. [EMBO Mol Med]
Abstract | Press Release

Gene Therapy of Endometriosis Introduced by Polymeric Micelles with Glycolipid-Like Structure
To reduce the side effects and improve the lack of clinical treatment countermeasures in endometriosis chemotherapy, a polymeric micelle gene delivery system composed of lipid grafted chitosan micelles and the pigment epithelium derived factor was designed. [Biomaterials] Abstract

Influence of Cationic Lipid Composition on Gene Silencing Properties of Lipid Nanoparticle Formulations of siRNA in Antigen-Presenting Cells
Scientists examined the uptake, intracellular trafficking, and gene silencing potency in primary bone marrow macrophages and dendritic cells of siRNA formulated in lipid nanoparticles containing four different ionizable cationic lipids namely DLinDAP, DLinDMA, DLinK-DMA, and DLinKC2-DMA. [Mol Ther]
Abstract | Press Release

A New Methodological Sequence to Expand and Transdifferentiate Human Umbilical Cord Blood Derived CD133+ Cells into a Cardiomyocyte-Like Phenotype
In this study researchers investigated optimizing a protocol for the expansion and subsequent differentiation of human umbilical cord blood derived CD133+ stem cells into a cardiomyocyte-like lineage. [Stem Cell Rev] Abstract | Press Release

High-Resolution Imaging and Antitumor Effects of GFP+ Bone Marrow-Derived Cells Homing to Syngeneic Mouse Colon Tumors
Investigators have established a mouse model for imaging bone marrow-derived cells from whole tumor to single-cell resolution, whereby the bone marrow of lethally irradiated host animals is reconstituted with EGFP-expressing bone marrow cells from matched TgActb(EGFP) donors. [Am J Pathol] Abstract | Press Release

Reprogramming within Hours Following Nuclear Transfer into Mouse but Not Human Zygotes
Using fertilized eggs, investigators found that when the zygotic genome was replaced with that of a somatic cell, development progressed normally throughout the cleavage stages, but then arrested before the morula stage. [Nat Commun] Abstract | Press Release

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SCIENCE NEWS
Idera Pharmaceuticals Presents Preclinical Data of Its Gene-Silencing Oligonucleotides at Cell Symposium on Regulatory RNAs
Idera Pharmaceuticals, Inc. presented new data on its novel gene-silencing oligonucleotide technology. [Press release from Idera Pharmaceuticals, Inc discussing research presented at the Cell Symposium on Regulatory RNAs, Chicago] Press Release

By Reprogramming Skin Cells Into Brain Cells, Scientists Gain New Insights Into Mental Disorders
Using skin cells from patients with mental disorders, scientists are creating brain cells that are now providing extraordinary insights into afflictions like schizophrenia and Parkinson’s disease. [ScienceDaily] Press Release

Clinical Trial Demonstrates Safety and Efficacy of Wound-Healing Treatment
Kerecis has successfully completed the first human clinical trial of its MariGen Lipid Collagen Matrix. [Kerecis] Press Release

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POLICY
Government of Canada Strengthens Cancer Research in British Columbia
Cancer research in British Columbia (BC) received a significant boost, thanks to nearly $2.2 million in federal funding announced for the BC Cancer Agency. [Government of Canada, Canada] Press Release

GAO Says White House Broke the Law by Holding Science Meetings with China
The watchdog agency for Congress has sided with an influential House of Representatives Republican in his attempt to block bilateral scientific exchanges with China. [Government Accountability Office (GAO), United States] Press Release

Russian Scientists Rally to Protest Funding Freeze
Several hundred researchers, many wearing lab coats, rallied to protest a funding freeze at Russia’s two grant organizations and on procurement regulations that they say are major obstacles for research. [Russian Academy of Science, Russia] Press Release

Brazilians Lured Back Home with Research Funding and Stability
With Brazil’s economy taking off and the country’s academic centers on the rise, more and more young scientists who went abroad for their training are toying with the idea of continuing their careers back home. [Brazilian Government, Brazil] Press Release

In Brazil, Basic Stem Cell Research Lags Behind Clinical Trials
To remedy the lag in basic stem cell science, the Brazilian government introduced several measures to give the stem cell field a boost. [Brazilian Government, Brazil] Press Release

New Framework Needed to Thwart Brazil’s Crippling Bureaucracy
To eliminate some of the red tape that has been blamed for stultifying research and innovation, an expert panel representing state funding agencies and science secretaries released a report on 26 August calling for a new code of conduct governing how scientific research is carried out in Brazil. [National Council of State Research Funding Foundations, Brazil] Press Release


BUSINESS

Maryland Stem Cell Research Commission Issues Requests for Applications
Per the Maryland Stem Cell Commission (Commission) meeting on Sept.15, 2010, the Commission has issued three official Requests for Applications for funding through the Maryland Stem Cell Research Fund under the Maryland Stem Cell Research Act. [Maryland Stem Cell Research Fund] Press Release

Genesis Biopharma Signs Worldwide License Agreement with the NIH for Adoptive Cell Therapy to Treat a Variety of Cancers
Genesis Biopharma, Inc. announced it has entered into a Patent License Agreement with the National Institutes of Health (NIH). [Genesis Biopharma, Inc.] Press Release

Fred Hutchinson Cancer Research Center Breaks Ground for First Collaborative, Comprehensive Cancer Center in Sub-Saharan Africa
A pioneering international collaboration forged by Fred Hutchinson Cancer Research Center together with the Uganda Cancer Institute has broken ground for the future construction of a state-of-the-art cancer training and outpatient treatment facility in Kampala. [Fred Hutchinson Cancer Research Center] Press Release

MU Signs Academic Agreement with K.S.R. Educational Institutions of India
University of Missouri (MU) officials announced a five-year agreement with K.S.R. Educational Institutions of India that will promote advanced research and training in nanomedicine and nanotechnology. [University of Missouri] Press Release

British Scientist Working to Cure Common Form of Blindness Named Recipient of Inaugural NYSCF-Robertson Prize in Stem Cell Research
The New York Stem Cell Foundation (NYSCF) announced that a British stem cell scientist working to cure blindness will be the first recipient of The New York Stem Cell Foundation – Robertson Prize in Stem Cell Research, a $200,000 prize awarded annually for extraordinary achievement in translational stem cell research. [The New York Stem Cell Foundation] Press Release

Carnegie’s Christoph Lepper Receives Prestigious Early Independence Award
Christoph Lepper is one of 10 recipients of the National Institutes of Health Director’s Early Independence Awards. [Carnegie Institution for Science] Press Release

Regenerating Eyes Using Cells From Hair
A young scientist who led research into the use of stem cells from hair follicles to treat the ocular surface disease has been named the winner of the Young Investigator Award by the journal STEM CELLS. [Friedrich-Alexander University of Erlangen-Nürnberg] Press Release

New Swedish Fellowship Program Aims to Give Young Researchers a Leg Up
The Wallenberg Academy Fellows program, announced by the Knut and Alice Wallenberg Foundation in Stockholm, will give generous start-up funds and access to positions in Swedish universities to 125 grantees over the next 5 years. [ScienceInsider] Press Release

Penn Researchers Receive $9 Million NIH Grant to Study Relationship Between Gene Variants and Cardiovascular Disease
Daniel J. Rader, MD, Edward Morrisey, PhD, and researchers at the Medical College of Wisconsin received a five-year, $9 million grant for stem cell research from the National Institutes of Health (NIH)’s National Human Genome Research and the National Heart Lung and Blood Institutes. [Penn Medicine] Press Release

Neuralstem Receives Notice of Patent Allowance for Spinal Cord Delivery Platform
Neuralstem, Inc. announced that it has received a notice of allowance from the U.S. Patent and Trademark Office for patent application 12/418,170 pertaining to a “Spinal Platform and Method for Delivering a Therapeutic Agent to a Spinal Cord Target.” [Neuralstem, Inc.] Press Release

Nuvilex Advances Studies Combining Its Encapsulation and Live Stem Cell Technologies
Nuvilex, Inc. announced substantial testing has been completed and more testing is underway combining its patented encapsulation technology with live stem cells. [Nuvilex, Inc] Press Release



NIH
NIH Grant Success Rate Likely Hit Historic Low in 2011
Grant success rates at the National Institutes of Health (NIH) appear to have plunged to an all-time low in 2011. ScienceInsider has learned that an early estimate from the NIH Office of Extramural Research puts the success rate for research grants at 17.4% for the fiscal year that ended 30 September. Press Release

ENCODE RFAs Expand Effort to Understand the Genome
On Tuesday, October 4, 2011, the National Human Genome Research Institute released three new Requests for Applications (RFAs) in which it announced plans to invest more than $120 million over four years (or about $31.5 million in FY 2012) to expand ENCODE (ENcyclopedia Of DNA Elements) research. Press Release

NIH Institute Director Receives Distinguished Achievement Award
Roderic Pettigrew, Ph.D., M.D., at the National Institutes of Health has been selected to receive the 2011 Distinguished Achievement Award from the Biomedical Engineering Society. Press Release

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2011-26222)

National Center For Research Resources; Notice of Closed Meeting (FR Doc No: 2011-26218)

National Institute of General Medical Sciences; Notice of Closed Meeting (FR Doc No: 2011-26206)

Office of Biotechnology Activities; Recombinant DNA Research: Action Under the NIH Guidelines for Research Involving Recombinant DNA Molecules (NIH Guidelines) (FR Doc No: 2011-26224)

Request for Information (RFI): To Solicit Input on Partnerships between Extramural and NIH Intramural Investigators Utilizing the NIH Clinical Center (NOT-OD-12-005)

The NEI Mentored Clinical Scientist Development Program Award (K12) (PAR-12-002)

Update of NIH Late Application Policy to Reflect Change in Due Dates for New Investigator R01 Resubmission Applications (NOT-OD-12-001)


CBER
2011 Biological License Application Supplement Noteworthy Approvals

2011 Biological Device Application Approvals

Biological Product Deviation Reporting and HCT/P Deviation Reporting – Deviation Codes

Biological Product Deviation Reporting and HCT/P Deviation Reporting – Non-Blood Product Codes


REGULATORY
Food and Drug Administration (United States)

Senators Propose Relaxing FDA Conflict Rules
A bill that would loosen conflict of interest rules for advisers to the Food and Drug Administration has been proposed by three U.S. senators seeking to speed up review times for medical devices. Press Release

Pilot Program for Parallel Review of Medical Products [Docket No. FDA-2010-N-0308]


EVENTS
NEW Educational Webinar on Mouse Phenome Database
October 20, 2011
Webinar

NEW Cell Therapy Approaches for Lung Diseases
October 24, 2011
London, United Kingdom

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


JOB OPPORTUNITIES

Lab Technologist – Tissue Culture (STEMCELL Technologies)

Research and Development Technologist, hPSC Media (STEMCELL Technologies)

Scientist (iPSC) (STEMCELL Technologies)

Contract Assay Service Technologist (STEMCELL Technologies)

Lab Technologist – Pluripotent Stem Cells (STEMCELL Technologies)

Quality Control Technologist – Cell Separation (STEMCELL Technologies)

Scientist – Pluripotent Stem Cells (STEMCELL Technologies)

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

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

Lab Technologist Position (Michigan State University)

Postdoctoral Fellow – Islet Cell Electrophysiology (Vanderbilt University)

Research Scientist (Vaccine/Bio-Process/Cell Therapy) (LG LIFE SCIENCE)


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