Volume 13.15 | Apr 23

Issue 13.15, April 23, 2012
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In Vivo Reprogramming of Murine Cardiac Fibroblasts into Induced Cardiomyocytes
Researchers used genetic lineage tracing to show that resident non-myocytes in the murine heart can be reprogrammed into cardiomyocyte-like cells in vivo by local delivery of Gata4, Mef2c and Tbx5 after coronary ligation. [Nature] Abstract | Press Release

Complimentary Wallchart: Human Hematopoietic Progenitors. Request Your Copy Today.

PUBLICATIONS (Ranked by Impact Factor of the Journal)

Restoration of Vision After Transplantation of Photoreceptors
The authors showed that transplanted rod precursors form classic triad synaptic connections with second-order bipolar and horizontal cells in the recipient retina. The newly integrated photoreceptor cells are light-responsive with dim-flash kinetics similar to adult wild-type photoreceptors. [Nature] Abstract | Press Release

RNA Interference Inhibits DUX4-Induced Muscle Toxicity In Vivo: Implications for a Targeted FSHD Therapy
Investigators tested a preclinical RNA interference-based DUX4 gene silencing approach as a prospective treatment for facioscapulohumeral muscular dystrophy (FSHD). [Mol Ther] Abstract

Alteration of Cardiac Progenitor Cell Potency in Golden Retriever Muscular Dystrophy Dogs
Researchers provide essential information that canine cardiac progenitor cells may be used to alleviate cardiac involvement in large preclinical model 
of duchenne muscular dystrophy. [Cell Transplant] Abstract

Generation of Kidney from Pluripotent Stem Cells via Blastocyst Complementation
Researchers used blastocyst complementation to generate pluripotent stem cell-derived donor organs in vivo. [Am J Pathol] Abstract

Differentiation and Transplantation of Functional Pancreatic Beta Cells Generated from Induced Pluripotent Stem Cells Derived from a Type 1 Diabetes Mouse Model
Researchers demonstrated a proof of concept for cellular therapy using non-obese diabetic mouse-derived induced pluripotent stem cells. [Stem Cells Dev] Abstract

Comparative Study on the Therapeutic Potential of Neurally Differentiated Stem Cells in a Mouse Model of Multiple Sclerosis
Using a mouse model of multiple sclerosis, scientists evaluated the therapeutic potential of neural stem cells (NSCs) derived from embryonic stem cells by two different neural differentiation protocols that utilized adherent culture conditions and compared their effect to primary NSCs derived from the subventricular zone. [PLoS One] Abstract

Cell Therapy Limits Myofibroblast Differentiation and Structural Cardiac Remodeling: bFGF-Mediated Paracrine Mechanism
The effect of cell therapy on post-infarction cardiac fibroblast and extracellular matrix regulation was examined. [Circ Heart Fail] Abstract

Ex Vivo Functional Analysis, Expansion and Adoptive Transfer of Cytomegalovirus-Specific T-Cells in Patients with Glioblastoma Multiforme
Researchers found that adoptive transfer of in vitro-expanded cytomegalovirus-specific CD8+ T-cells in combination with temozolomide therapy into a patient with recurrent glioblastoma multiforme was coincident with a long-term disease-free survival. [Immunol Cell Biol] Abstract

Targeted Delivery of PLK1-siRNA by ScFv Suppresses Her2+ Breast Cancer Growth and Metastasis
Scientists tested whether Polo-like kinase 1 (PLK1) small interfering RNAs (siRNAs) complexed with a Her2-single-chain fragmented antibody (ScFv)-protamine peptide fusion protein could suppress Her2+ breast cancer cell lines and primary human cancers in orthotopic breast cancer models. [Sci Transl Med]
Abstract | Press Release

Patient-Specific Induced Pluripotent Stem Cells as a Model for Familial Dilated Cardiomyopathy
Researchers generated cardiomyocytes from induced pluripotent stem cells derived from patients in a dilated cardiomyopathy family carrying a point mutation in the gene encoding sarcomeric protein cardiac troponin T. [Sci Transl Med] Abstract | Press Release

Fully Functional Hair Follicle Regeneration through the Rearrangement of Stem Cells and Their Niches
Investigators demonstrated fully functional hair organ regeneration via the intracutaneous transplantation of a bioengineered pelage and vibrissa follicle germ. [Nat Commun] Abstract | Press Release

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Alnylam Reports Positive Clinical Results for ALN-PCS, an RNAi Therapeutic Targeting PCSK9 for the Treatment of Severe Hypercholesterolemia
Alnylam Pharmaceuticals, Inc. announced positive results from its Phase I clinical trial of ALN-PCS. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the American Heart Association’s Arteriosclerosis, Thrombosis and Vascular Biology 2012 Scientific Sessions, Chicago] Press Release

ACT Announces Third Dry AMD Patient Treated in Clinical Trial
Advanced Cell Technology, Inc. (ACT) announced the dosing of the third patient in its Phase I/II trial for dry age-related macular degeneration (dry AMD) using retinal pigment epithelial cells derived from human embryonic stem cells. The outpatient transplantation surgery was performed successfully, and the patient is recovering uneventfully. [Advanced Cell Technology, Inc.] Press Release

Oxford BioMedica Announces Successful Completion of ProSavin® Phase I/II Study in Parkinson’s Disease
Oxford BioMedica plc announced that it has successfully completed a Phase I/II study to assess the safety, efficacy and dose evaluation of ProSavin® in patients with mid-stage Parkinson’s disease who are experiencing reduced benefit on L-DOPA “equivalent” therapy. [Oxford BioMedica plc] Press Release

TiGenix Rheumatoid Arthritis Phase IIa Study Successfully Passes Last Safety Hurdle
TiGenix announced that upon review of the safety data of the first three patients of the third cohort of the company’s Phase IIa clinical trial in rheumatoid arthritis (Cx611) it has received the go-ahead from the independent Safety Monitoring Board to recruit and dose the remaining patients of this cohort. [TiGenix NV] Press Release

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Syrian Uprising Takes Toll on Scientific Community
The civil unrest that erupted in Syria in March last year has left the country’s scientific community in turmoil, researchers say. Heavy cuts have been made to research budgets, and work at the majority of Syria’s universities and research centers has ground to a halt. [Syrian Government, Syria] Press Release

House Panel Tops Senate Mark for NSF
A House of Representatives spending panel wants to nearly match the president’s budget request for the National Science Foundation (NSF). [House of Representatives, United States] Press Release

NSF Budget Year Starts Out Well
The National Science Foundation (NSF) would receive a 3.3% budget increase next year under a bill taken up by a Senate spending panel. Although the $240 million increase falls $100 million short of the president’s request for the $7 billion agency, it still represents a remarkable show of support for the basic research agency, given that the panel had $1 billion less to work with this year. [National Science Foundation, United States]
Press Release

World Gene Therapy Congress Europe 2012


Penn State Hershey and Serametrix to Seek Companion Diagnostic for Immunotherapy in Pediatric Cancers
Penn State Hershey Children’s Hospital and Serametrix will collaborate on developing a diagnostic test for predicting individual clinical response to a novel immune therapy for brain cancer. [Serametrix Corporation] Press Release

UTHealth Uses $1.9 Million Grant to Study Therapy for Autoimmunity
A researcher at The University of Texas Health Science Center at Houston (UTHealth) is studying a novel cell therapy that could help avoid autoimmune problems after stem cell transplantation, as well as potentially treat other autoimmune diseases. [The University of Texas Health Science Center at Houston] Press Release

Human Genome Sciences Announces Unsolicited Offer from GlaxoSmithKline; HGS Board of Directors Authorizes Exploration of Strategic Alternatives
Human Genome Sciences, Inc. (HGS) announced that it has received an unsolicited proposal from GlaxoSmithKline plc to acquire HGS for $13.00 per share in cash. [Human Genome Sciences, Inc.]
Press Release

Dr Barry Snow to Lead Parkinson’s Trial for Living Cell Technologies
Living Cell Technologies Limited announced that Dr Barry Snow has agreed to be the Principal Investigator for its Phase I clinical trials of NTCELL for Parkinson’s disease. [Living Cell Technologies Limited] Press Release

New SSEA-5 Antibody Launched for Stem Cell Research
GeneTex is set to launch a new monoclonal antibody against stage-specific embryonic antigen 5 (SSEA-5), a marker for human pluripotent stem cells that may also improve the safety of stem cell-based treatments. [GeneTex Inc.] Press Release

Marina Biotech Regains Intellectual Property Rights to Its tkRNAi(TM) Technology for Agriculture and Veterinary Uses
Marina Biotech, Inc. announced that it has regained intellectual property rights to its transkingdom RNAi (tkRNAi) drug discovery platform for both agriculture and veterinary uses. [Marina Biotech, Inc.] Press Release

Baxter and Halozyme Provide Update on HyQ Biologics License Application
Baxter International Inc. and Halozyme Therapeutics, Inc. announced that the U.S. Food and Drug Administration is requesting additional information to complete its review of the HyQ Biologics License Application. [Halozyme Therapeutics, Inc.] Press Release

Pluristem Receives U.S. FDA Clearance for Phase II Clinical Trial in Intermittent Claudication
Pluristem Therapeutics, Inc. announced that the United States Food and Drug Administration (FDA) has granted the company clearance to start a Phase II clinical trial using the company’s PLX-PAD cell product candidate for the treatment of intermittent claudication. [Pluristem Therapeutics, Inc.] Press Release

Organogenesis Inc. Announces Major Milestones in Improved Apligraf® Reimbursement and Access to Care for Patients Suffering from Chronic Wounds
Organogenesis Inc. announced that Apligraf® has established improved coverage from multiple payors, improving access to care for millions of patients suffering from chronic, non-healing wounds. [Organogenesis Inc.] Press Release

NewLink Genetics Receives Notice of Allowance from the Japan Patent Office for New Patent Broadly Covering Its HyperAcute(R) Cancer Immunotherapy Products
NewLink Genetics Corporation announced that the Japan Patent Office has issued a notice of allowance for a patent entitled “Antitumor Vaccination Using Allogeneic Tumor Cells Expressing Alpha (1,3)-Galactosyltransferase”, which contains broad pharmaceutical composition claims covering NewLink’s HyperAcute products for the treatment of cancer. [NewLink Genetics Corporation] Press Release

Intervertebral Disc Repair Phase II Trial Update
Mesoblast Limited announced that its Phase II clinical trial investigating the use of allogeneic, or off-the-shelf, mesenchymal precursor cells for non-surgical restoration of degenerated intervertebral discs and treatment of low back pain has enrolled 50% of the total study patients. [Mesoblast Limited] Press Release

New Research Center Unveiled at Annual Ohio State Conference
The Ohio State University has established a partnership of experts who will work together to discover novel treatments to accelerate wound healing and rehabilitation for patients. Ohio State’s Center for Regenerative Medicine and Cell-Based Therapies was created to further develop the research programs in these respective fields. [The Ohio State University Wexner Medical Center] Press Release


With Flat Budget Proposed, Clamor Builds in Congress for More NIH Spending
Two bipartisan “Dear Colleague” letters circulating in Congress have drawn many more signatures than most. Their message is that NIH spending should grow well above the current fiscal year’s level. Press Release

Clinical Sequencing Exploratory Research (U01) (RFA-HG-12-009)

Clinical Sequencing Exploratory Research Coordinating Center (U01) (RFA-HG-12-008)

2012 Biological License Application Approvals


Food and Drug Administration (United States)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Guidance for Clinical Trial Sponsors: Establishment and Operation of Clinical Trial Data Monitoring Committees (FR Doc No: 2012-9533)

Agency Information Collection Activities; Announcement of Office of Management and Budget Approval; Current Good Manufacturing Practices and Related Regulations for Blood and Blood Components; and Requirements for Donor Testing, Donor Notification, and “Lookback” (FR Doc No: 2012-9531)

Food and Drug Administration Patient Network Annual Meeting; Input Into Food and Drug Administration Benefit-Risk Decisionmaking: Opportunities and Challenges; Hosted by the Food and Drug Administration Office of Special Health Issues; Notice of Meeting (FR Doc No: 2012-9418)

European Medicines Agency (European Union)

European Medicines Agency Maintains Recommendation Not to Grant a Marketing Authorization for Glybera
The European Medicines Agency’s Committee for Medicinal Products for Human Use maintained its recommendation not to grant a marketing authorization for the orphan medicine Glybera (alipogene tiparvovec), from Amsterdam Molecular Therapeutics B.V. Press Release


NEW The Jackson Laboratory: Mouse Phenome Database – Knowledge to Drive Discovery
April 26, 1:00PM (ET in US)

NEW Stem Cells USA & Regenerative Medicine Congress 2012
September 20-21, 2012
Boston, United States

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


Scientist – Induced Pluripotent Stem Cells (STEMCELL Technologies, Inc.)

Scientist – Epithelial Cell Research (STEMCELL Technologies, Inc.)

Scientist – hPSC (STEMCELL Technologies, Inc.)

Scientist – hPSC Bioengineering (STEMCELL Technologies, Inc.)

Scientist – Antibody and Hybridoma Technology Platforms (STEMCELL Technologies, Inc.)

Scientist – Endothelial Cells (STEMCELL Technologies, Inc.)

Business Analyst – Product Management (STEMSOFT Software, Inc.)

Senior Development Associate (Genzyme Corporation)

Postdoctoral Position in Stem Cell Biology/Neuroscience/Cancer (The Jackson Laboratory)

Associate Director, Cell Therapy (Seattle Cancer Care Alliance)

iPSC Scientist Position – Laboratory of Translational Cell Biology (The Bassell Lab, Emory University)

Postdoctoral Position – Translational Breast Cancer Research (ISREC/EPFL)

Senior Scientist (Techshot, Inc.)

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