Volume 14.12 | Apr 1

Issue 14.12 April 1, 2013
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Treatment of HCV Infection by Targeting MicroRNA
Miravirsen is a locked nucleic acid-modified DNA phosphorothioate antisense oligonucleotide that sequesters mature microRNA-122 in a highly stable heteroduplex, thereby inhibiting its function. In this phase IIa study at seven international sites, researchers evaluated the safety and efficacy of miravirsen in 36 patients with chronic hepatitis C virus (HCV) genotype 1 infection. [N Engl J Med] Full Article | Press Release

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

PUBLICATIONS (Ranked by impact factor of the journal)

Drug Screening Using a Library of Human Induced Pluripotent Stem Cell-Derived Cardiomyocytes Reveals Disease Specific Patterns of Cardiotoxicity
Investigators generated a library of human induced pluripotent stem cell-derived cardiomyocytes from patients with various hereditary cardiac disorders to model differences in cardiac drug toxicity susceptibility for patients of different genetic backgrounds. [Circulation] Abstract | Press Release

Reducing TMPRSS6 Ameliorates Hemochromatosis and β-Thalassemia in Mice
Researchers identified second generation antisense oligonucleotides (ASOs) targeting mouse Tmprss6. ASO treatment in mice affected by hemochromatosis significantly decreased serum iron, transferrin saturation and liver iron accumulation. Furthermore, ASO treatment of mice affected by β-thalassemia decreased the formation of insoluble membrane-bound globins, ROS, and apoptosis, and improved anemia. These animals also exhibited lower erythropoietin levels, a significant amelioration of ineffective erythropoiesis and splenomegaly, and an increase in total hemoglobin levels. [J Clin Invest] Full Article | Press Release

Transgenic Overexpression of Ribonucleotide Reductase Improves Cardiac Performance
Researchers reported the effect of chronically enhanced 2-deoxy-ATP (dATP) concentration on cardiac function using a transgenic mouse that overexpresses the enzyme ribonucleotide reductase, which catalyzes the rate-limiting step in de novo deoxyribonucleotide biosynthesis. The data are sufficiently compelling to consider elevated cardiac [dATP] as a therapeutic option to treat systolic dysfunction. [Proc Natl Acad Sci USA]
Abstract | Press Release

Bone Marrow Injection Stimulates Hepatic Ductular Reactions in the Absence of Injury via Macrophage-Mediated TWEAK Signaling
In various organs, bone marrow cell (BMC) therapy has shown promising preliminary results, but to date no definite mechanism has been demonstrated to account for the observed benefit in organ regeneration. Researchers examined a model without injury; a single i.v. injection of unfractionated BMCs in healthy mice. [Proc Natl Acad Sci USA] Abstract | Full Article

Long-Term Follow-Up of Foamy Viral Vector-Mediated Gene Therapy for Canine Leukocyte Adhesion Deficiency
Investigators reported the long-term follow-up (4-7 years) of four dogs with canine leukocyte adhesion deficiency treated with foamy viral vector-mediated gene therapy. [Mol Ther] Abstract

Adeno-Associated Virus Serotype 9 Efficiently Targets Ischemic Skeletal Muscle following Systemic Delivery
As adeno-associated virus serotype 9 (AAV-9) transduces skeletal muscle at high efficiency following systemic delivery, researchers employed AAV-9 vectors bearing luciferase or enhanced green fluorescent protein reporter genes to test the hypothesis that increased desialylation of cell-surface glycans secondary to hindlimb ischemia might help offset the reduction in tissue perfusion that occurs in mouse models of peripheral arterial disease. [Gene Ther] Abstract

Papilla Regeneration by Injectable Stem Cell Therapy with Regenerative Medicine: Long-Term Clinical Prognosis
Researchers aimed to determine the possibility and long-term prognosis of novel papilla regeneration with regenerative medicine, i.e. tissue-engineered papilla, and to investigate the potential of a tissue-engineering method for soft-tissue augmentation, especially aesthetic improvement of black triangle, with mesenchymal stem cells as the isolated cells, platelet-rich plasma as the growth factor and hyaluronic acid as the scaffold. [J Tissue Eng Regen Med] Abstract

Transplantation of Bone Marrow-Derived MSCs Improves Cisplatinum-Induced Renal Injury through Paracrine Mechanisms
Mesenchymal stem cells (MSCs) have been reported to preserve renal function in various models of acute kidney injury (AKI). Different routes were used to transplant MSCs but the role of cell transplantation routes in directing outcomes has been unknown. Researchers evaluated organ bio-distributions of transplanted MSCs, and correlated survival of transplanted cells with outcomes in mice with cisplatinum-induced AKI. [Exp Mol Pathol] Abstract

Gain Hands-On Training. New Course: Differentiation of hPSCs to Neural Progenitor Cells

Advances in Stem Cell Therapy against Gliomas
The authors discuss current trends and the latest developments in stem cell therapy against malignant gliomas from both the experimental laboratory and the clinic. [Trends Mol Med] Abstract

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research field.

New StemSpan(TM) SFEM II - for optimal CD34+ cell expansion of bone marrow and cord blood cells


Dosing Completed in ReNeuron’s Phase I Clinical Trial in Stroke
Following a further positive assessment from the independent Data Safety Monitoring Board for the earlier study, the final dose cohort in the ongoing PISCES Phase I clinical trial with ReN001 has now been treated and the final patient dosed in the study has been discharged from hospital. [ReNeuron Group plc] Press Release

Tengion Receives MPA Approval of CTA for Neo-Kidney Augment Phase I Trial in Sweden
Tengion, Inc. announced the acceptance of the Company’s Clinical Trial Application (CTA) filed with the Medical Products Agency (MPA) in Sweden to initiate a Phase I clinical trial to evaluate the safety and delivery of its Neo-Kidney Augment™ product in up to five patients with advanced chronic kidney disease. [Tengion, Inc.]
Press Release


Stem-Cell Ruling Riles Researchers
Italy’s health minister, Renato Balduzzi, has decreed that a controversial stem-cell treatment can continue in 32 terminally ill patients, mostly children – even though the stem cells involved are not manufactured according to Italy’s legal safety standards. [Italian Health Minister, Italy] Press Release

Chair of House Spending Panel Says NIH Grants Violate Rules against Lobbying
The chair of the House of Representatives spending panel that funds the National Institutes of Health (NIH) is claiming that some federally funded biomedical research violates government restrictions on lobbying state lawmakers or Congress. [House of Representatives, United States] Press Release


Mount Sinai Leads Global Program Using Stem Cells, Collaborates with The New York Stem Cell Foundation to Accelerate Cures for Alzheimer’s Disease
Sam Gandy, MD, PhD, of the Icahn School of Medicine at Mount Sinai is leading an international team of researchers working to reprogram skin cells into brain cells to gain a better understanding of Alzheimer’s disease. As part of the Consortium, Dr. Gandy is collaborating with Scott Noggle, PhD, the NYSCF – Charles Evans Senior Research Fellow for Alzheimer’s Disease and Director of the New York Stem Cell Foundation (NYSCF)’s laboratory in Manhattan. [New York Stem Cell Foundation] Press Release

CDI Partners with Buck to Derive and Bank iPSCs
Cellular Dynamics International (CDI) announced that the company is opening a facility in Novato, Calif., leasing space from the Buck Institute for Research on Aging (Buck). This opening of a second CDI facility is in response to the company receiving a $16 million grant award from the California Institute of Regenerative Medicine to create and bank human induced pluripotent stem cells (iPSCs). [Buck Institute for Research on Aging]
Press Release

Retina Institute Japan and Dainippon Sumitomo Pharma Form a Capital Alliance towards the Development of Nobel Prize Winning iPS Cell Technology
Retina Institute Japan K.K. (RIJ) and Dainippon Sumitomo Pharma Co., Ltd. (DSP) announced that the two companies signed an agreement that DSP invests into RIJ to discuss alliance to put iPS cell technology to practical use. [Dainippon Sumitomo Pharma Co., Ltd.] Press Release

ReNeuron and Cell Therapy Catapult in Landmark Collaboration on New Cell Manufacturing Technologies – First UK Corporate Deal for CT Catapult to Focus on ReNeuron’s Lead Cell Therapy Candidate
ReNeuron and the Cell Therapy Catapult announced that they have signed an agreement to work together on new cell therapy manufacturing technologies and assays. [ReNeuron Group plc] Press Release

AstraZeneca and Moderna Therapeutics Announce Exclusive Agreement to Develop Pioneering Messenger RNA Therapeutics™ in Cardiometabolic Diseases and Cancer
AstraZeneca announced an exclusive agreement with Moderna Therapeutics to discover, develop and commercialize pioneering messenger RNA therapeutics™ for the treatment of serious cardiovascular, metabolic and renal diseases as well as cancer. [AstraZeneca] Press Release

NeoStem Awarded Second Year of NIAID Research Grant for the Development of VSEL Technology for Radiation Exposure
NeoStem, Inc. announced that it has been awarded the second year of a two year grant totaling $595,252 for the “Development of Human, Autologous, Pluripotent Very Small Embryonic Like (VSELs) Stem Cells as a Countermeasure to Radiation Threat”, Grant Number 5R43AI098325-02 from the National Institute of Allergy and Infectious Diseases (NIAID), a division of the National Institutes of Health. [NeoStem, Inc.] Press Release

Harper Government Announces New Personalized Health Research Projects Across Canada
Joy Smith on behalf of the Honorable Leona Aglukkaq, Minister of Health, announced an investment of $13.7 million to support four research projects in Ontario. The announcement was made during a visit to the Children’s Hospital of Eastern Ontario (CHEO) in Ottawa to learn more about a project, under Dr. Kym Boycott’s leadership, that will use new technologies to identify the genes implicated in many rare disorders and test treatment against the identified genes. [CHEO Research Institute] Press Release

Regenerative Medicine Company, Eqalix, Announces Grant from Nanotechnology Institute in Philadelphia, Pennsylvania
Eqalix has announced they have received a $50,000 grant from the Nanotechnology Institute in Philadelphia Pennsylvania which will facilitate the advancement of the development of its technologies for regenerative medicine. [The Nanotechnology Institute] Press Release

ReNeuron Receives Regulatory and Ethical Approvals to Commence Clinical Trial in UK with Stem Cell Therapy Candidate for Critical Limb Ischemia
ReNeuron announced that it has received regulatory and ethical approvals to commence a Phase I clinical trial in the UK with its ReN009 stem cell therapy program targeting the major unmet medical need, critical limb ischemia. [ReNeuron Group plc] Press Release

Cytori Patents Method of Treating Renal Diseases Using Adipose-Derived Regenerative Cells
Cytori Therapeutics received US Patent No. 8,404,229 (the ‘229 patent) for methods of treating renal diseases using adipose-derived regenerative cells (ADRCs). The ‘229 patent covers treatment of a broad range of renal disorders, including acute kidney disease and chronic kidney disease. The ‘229 patent also covers multiple means of delivering the ADRCs, including to the kidney directly, to the renal vasculature or to the renal parenchyma. [Cytori Therapeutics] Press Release


Government-Owned Inventions; Availability for Licensing (FR Doc No: 2013-06836)

Center for Scientific Review; Notice of Closed Meeting (FR Doc No: 2013-06806)

Prospective Grant of Exclusive License: Papilloma Pseudovirus and Virus-Like Particles as a Delivery System for Human Cancer Therapeutics and Diagnostics (FR Doc No: 2013-06837)

Prospective Grant of Start-Up Exclusive License: Photosensitizing Antibody-Fluorophore Conjugates for Photoimmunotherapy (FR Doc No: 2013-07166)


Food and Drug Administration (United States)

Draft Guidance for Industry on Formal Meetings Between FDA and Biosimilar Biological Product Sponsors or Applicants; Availability (FR Doc No: 2013-07445)

Medicines and Healthcare Products Regulatory Agency (United Kingdom)

Fees for Manufacturer’s Licenses

Fees for Clinical Trials Applications


NEW The Jackson Laboratory: Humanized NSG Mice – An Innovative Platform for Advancing Preclinical Research
April 11, 1:00PM (ET in US)

NEW Cold Spring Harbor Laboratory: Mouse Development, Stem Cells & Cancer
June 5-25, 2013
Cold Spring Harbor, United States

NEW 14th World Congress of the International Pancreas and Islet Transplant Association (IPITA) 2013
September 24-27, 2013
Monterey, California

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


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

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

Cord Blood Laboratory Manager (Puget Sound Blood Center)

Postdoctoral Fellow – Regenerative Medicine (The University of Texas Health Science Center at Houston)

Senior Scientist – Stem Cell Therapy: Metabolic Diseases (Paramount Recruitment)

Postdoctoral Research Fellow – Immunology (Beth Israel Deaconess Medical Center, Harvard Medical School)

Postdoctoral Research Fellow – Regenerative Medicine (Loma Linda University)

Postdoctoral Research Associate (Washington State University)

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