Cell Therapy News Volume 13.08 | Mar 5 2012

May 12, 2022

Issue 13.08, March 5, 2012

TOP STORY

SERCA2a Gene Therapy Restores MicroRNA-1 Expression in Heart Failure via an Akt/FoxO3A-Dependent Pathway
Researchers studied the effects of therapeutic AAV9.SERCA2a (sarcoplasmic reticulum calcium ATPase 2a) gene therapy on cardiac miRNome expression and focused on regulation, expression, and function of miR-1 in reverse remodeled failing hearts. [Eur Heart J] Abstract

PUBLICATIONS (Ranked by Impact Factor of the Journal)

Signaling Network Crosstalk in Human Pluripotent Cells: A Smad2/3-Regulated Switch that Controls the Balance between Self-Renewal and Differentiation
In this report, investigators describe a signaling mechanism where PI3K/Akt activity maintains self-renewal by restraining prodifferentiation signaling through suppression of the Raf/Mek/Erk and canonical Wnt signaling pathways. [Cell Stem Cell] Abstract | Press Release

Genetic Regulators of a Pluripotent Adult Stem Cell System in Planarians Identified by RNAi and Clonal Analysis
Researchers utilized transcriptional profiling to identify planarian genes expressed in adult proliferating, regenerative cells. Researchers also developed quantitative clonal analysis methods for expansion and differentiation of adult pluripotent stem cell descendants that, together with RNAi, revealed gene roles in stem cell biology. [Cell Stem Cell] Abstract | Press Release

AMD1 Is Essential for ESC Self-Renewal and Is Translationally Down-Regulated on Differentiation to Neural Precursor Cells
Scientists describe a new role for Amd1 (adenosyl methionine decarboxylase), a key enzyme in the polyamine synthesis pathway, in regulating both embryonic stem cell (ESC) self-renewal and differentiation to the neural lineage. [Genes Dev] Abstract | Press Release

Cationic Nanohydrogel Particles as Potential siRNA Carriers for Cellular Delivery
In this work, a new concept of synthesizing polymeric cationic nanohydrogels was developed, which offers a promising strategy to complex and transport short, double-stranded RNA (siRNA) into cells. [ACS Nano] Abstract

A Potent Vaccination Strategy that Circumvents Lymphodepletion for Effective Anti-Tumor Adoptive T Cell Therapy
Here researchers describe an alternative approach to total body irradiation and high dose IL2 for optimizing adoptive cell therapy, resulting in dramatic therapeutic effects against established melanomas in mice. [Cancer Res] Abstract

Preclinical Safety and Efficacy of Human CD34⁺ Cells Transduced with Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome
Researchers evaluated the preclinical safety and efficacy of human CD34⁺ cells transduced with lentiviral vectors encoding Wiskott-Aldrich syndrome protein. [Mol Ther] Abstract

Dendritic Cells Loaded with mRNA Encoding Full-Length Tumor Antigens Prime CD4⁺ and CD8⁺ T Cells in Melanoma Patients
This work provides definitive proof that dendritic cells presenting the full antigenic spectrum of tumor antigens can induce T cells specific for novel epitopes and can be administered to patients irrespective of their human leukocyte antigen type. [Mol Ther] Abstract

The Use of Human Sweat Gland-Derived Stem Cells for Enhancing Vascularization during Dermal Regeneration
In this study, stem cells derived from human sweat glands were isolated, characterized, seeded in collagen scaffolds, and engrafted in a mouse full skin defect model for dermal regeneration. [J Invest Dermatol] Abstract

Transcutaneous Gene Gun Delivery of hNC16A Induces BPAG2-Specific Tolerance
In a skin grafting mouse model, scientists used gene gun transfection with a construct encoding hNC16A, the immunodominant domain of human bullous pemphigoid antigen 2 (hBPAG2), to induce antigen-specific immune tolerance. [J Invest Dermatol] Abstract

Intrarectal Administration of mCRAMP-Encoding Plasmid Reverses Exacerbated Colitis in Cnlp^−/− Mice
Here researchers sought to delineate the role of endogenous murine cathelicidin (mCRAMP) and the therapeutic efficacy of intrarectal administration of mCRAMP-encoding plasmid in ulcerative colitis using dextran sulfate sodium-challenged cathelicidin-knockout (Cnlp^−/−) mice as a model. [Gene Ther] Abstract

T Cells Redirected by a CD3ζ Chimeric Antigen Receptor Can Establish Self-Antigen-Specific Tumor Protection in the Long Term
Adoptive therapy with CD8⁺ T cells, which were redirected by a carcinoembryonic antigen (CEA)-specific, low-affinity chimeric antigen receptor with CD3ζ endodomain, eliminated CEA⁺ tumors in a primary response; cured mice produced an efficient recall response in the long term towards CEA⁺ tumor cells upon rechallenge. [Gene Ther] Abstract

Bystander Killing of Malignant Cells via the Delivery of Engineered Thymidine-Active Deoxycytidine Kinase for Suicide Gene Therapy of Cancer
Here investigators describe ‘suicide’ gene therapy of cancer based on the lentivirus-mediated delivery of a thymidine-active human deoxycytidine kinase variant. [Cancer Gene Ther] Abstract

Hydrogen Sulfide Increases Hepatic Differentiation in Tooth-Pulp Stem Cells
Researchers have recently established an experimental model of hepatic differentiation from human tooth-pulp stem cells using serum-free medium. The objective of the present study was to determine the effect of hydrogen sulfide on hepatic differentiation. [J Breath Res] Abstract | Press Release

SCIENCE NEWS

Marina Biotech Presents Advances in Oral Delivery of Nucleic Acids
Marina Biotech, Inc. reported data demonstrating the continued advancement of the Company’s clinical program for CEQ508, the only orally administered RNAi therapeutic in clinical development. Data were also presented on early research in the oral administration of single- and double-stranded nucleic acids. [Press release from Marina Biotech, Inc. discussing research presented at the AsiaTIDES, Oligonucleotide and Peptide(R) Research, Technology and Product Development Conference, Tokyo] Press Release

Baxter Initiates Phase III Adult Stem Cell Clinical Trial for Chronic Cardiac Condition
Baxter International Inc. announced that it has initiated a phase III pivotal clinical trial to evaluate the efficacy and safety of adult autologous CD34+ stem cells to increase exercise capacity in patients with chronic myocardial ischemia. [Baxter International Inc.] Press Release

LCT Completes Pre-Clinical Studies of NTCELL in Parkinson’s
Living Cell Technologies Limited (LCT) announced the successful completion of pre-clinical studies for NTCELL, a potential innovative treatment for Parkinson’s disease. [Living Cell Technologies Limited] Press Release

Gene Therapy Success in Third Immune Disease
Doctors report that five patients with x-CGD have responded to gene therapy, showing clear clinical benefit. [Great Ormond Street Hospital for Children NHS Foundation Trust] Press Release

UC San Diego Among First in Nation to Treat Brain Cancer with Novel Viral Vector
Researchers and surgeons are among the first in the nation to treat patients with recurrent brain cancer by directly injecting an investigational viral vector into their tumor. The injected Toca 511 is a retroviral replicating vector that is designed to deliver a cytosine deaminase gene selectively to cancer cells. [University of California (UC), San Diego] Press Release

POLICY

Another Bumper Year for Chinese Science
Central government spending on science and technology is slated to rise 12.4%, to 228.54 billion yuan ($36.23 billion). Scientists will also benefit from a 24% jump in funding for Project 985 and Project 211, which funnel money to elite universities. [National People’s Congress, China] Press Release

Budget Cuts Force Lay-Offs at United Nations Biotechnology Center
International Centre for Genetic Engineering and Biotechnology seeks extra support after main funder slashes contribution. The Italian Ministry of Foreign Affairs is cutting €2 million from its €12.4-million contribution, as part of a multi-billion-euro package of austerity measures approved by the Italian parliament last November. [International Centre for Genetic Engineering and Biotechnology] Press Release

Stem-Cell Therapy Takes Off in Texas
With Texas pouring millions of dollars into developing adult stem-cell treatments, doctors there are already injecting paying customers with unproven preparations, supplied by an ambitious new company. [NatureNews] Press Release

BUSINESS

Bioheart Announces University of Miami as Clinical Site for ANGEL Trial of LipiCell(TM)
Bioheart, Inc. announced that the company will conduct the ANGEL trial using adipose derived stem cell technology or LipiCell(TM) at the University of Miami Miller School of Medicine. [MarketWatch] Press Release

VistaGen Therapeutics and Duke University Enter Into Strategic Research Collaboration
VistaGen Therapeutics, Inc. and Duke University have entered into a strategic research collaboration aimed at combining their complementary expertise at the forefront of cardiac stem cell technology, electrophysiology and tissue engineering. The initial goal of the collaboration is to explore potential development of novel, engineered, stem cell-derived cardiac tissues to expand the scope of VistaGen’s drug rescue capabilities focused on heart toxicity. [VistaGen Therapeutics, Inc.] Press Release

Arrowhead Research Corporation and Axolabs GmbH Enter into Strategic Alliance and Master Services Agreement for RNAi Therapeutics
Arrowhead Research Corporation and Axolabs GmbH announced that they have entered into a Strategic Alliance and Master Services Agreement. Under the terms of the agreement, Axolabs will provide Arrowhead and its partners first-in-class oligonucleotide optimization, synthesis, and analytics. [Arrowhead Research Corporation] Press Release

Artificial Liver Cells Win Their Creator Prize for Their Potential to Reduce Animal Experiments
Producing liver cells that demonstrate inherited liver diseases from human skin cells has earned Dr Ludovic Vallier from the University of Cambridge a major prize from the National Centre for the Replacement, Refinement and Reduction of Animals in Research. [University of Cambridge] Press Release

QF Awards Top Stem Cell Researchers at International Conference
Research on preventing breast cancer recurrence, using organ regeneration to combat obesity-related diseases, and enabling vascular repair for patients suffering from cardiovascular disease received awards at the Qatar International Conference on Stem Cell Science and Policy 2012. [Qatar Foundation (QF)] Press Release

Clinical Development Program Receives FDA “Fast Track Designation” and Encouraging Phase Ib Safety Results for Viaskin Peanut
DBV Technologies announced that it has received “Fast Track Designation” for the Clinical Development Program of its Viaskin^® Peanut, a product designed for the treatment by Epicutaneous Immunotherapy of patients with peanut allergy. [DBV Technologies] Press Release

Arrowhead Announces Patent Issuance Covering siRNA Inhibitors of HIF-2a (EPAS1)
Arrowhead Research Corporation announced that the United States Patent and Trademark Office has issued U.S. Patent No. US 8,114,983 B2 entitled, “Compositions and use of EPAS1 inhibitors.” Included in the patent are claims directed to siRNA sequences targeting the hypoxia-inducible factor 2 alpha gene (HIF-2a) or endothelial PAS domain protein 1 (EPAS1), chemical modifications which enhance stability and/or biological activity, compositions, methods, and uses. [Arrowhead Research Corporation] Press Release

LCT Granted E.U. Patent for Tissue Engineering
Living Cell Technologies Limited (LCT) received formal notification that it has been granted E.U. Patent No. 1,438,395 from the European Patent Office. The patent protects the ability to culture two different types of cells derived from Auckland Island pigs, therefore enabling the assembly and maintenance of a mix of cells or artificial tissue in culture for xenotransplantation therapies. [Living Cell Technologies Limited] Press Release

Alnylam and the University of Massachusetts Medical School Announce Tuschl I Patent Upheld in European Opposition Proceedings
Alnylam Pharmaceuticals, Inc. and the University of Massachusetts Medical School reported that the European Patent Office has upheld the Tuschl I ’726 patent in oral opposition proceedings held in Munich, Germany. [Alnylam Pharmaceuticals, Inc.] Press Release

Tekmira Expands Product Pipeline with RNAi Therapeutic for the Treatment of Alcohol Dependence
Tekmira Pharmaceuticals Corporation announced that it is adding a new RNAi therapeutic targeting ALDH2 for the treatment of alcohol dependence to its product pipeline. [Tekmira Pharmaceuticals Corporation]
Press Release

Galena Biopharma Announces Expansion of Scientific Advisory Board
Galena Biopharma announced the appointment of David A. Scheinberg, M.D., Ph.D. to the Company’s Scientific Advisory Board. [Galena Biopharma] Press Release

International Cell Medicine Society Elects New President
The International Cellular Medicine Society (ICMS) announces that Dr Ricardo Rodriguez, MD has been elected President of the ICMS Board of Directors. [International Cellular Medicine Society] Press Release

NIH

Lawmakers Drop Proposal to Block NIH Public Access Policy
In a victory for open access advocates, two U.S. lawmakers yesterday dropped a bill that would have banned the National Institutes of Health (NIH) from requiring that its researchers submit their peer-reviewed manuscripts to a public archive. Press Release

Request for Information – Defining Unmet Needs for Clinical Translation of Cell-Based Therapies (NOT-RM-12-008)

Notice of Change in Participation of NIH Institutes and Centers in PA-10-138 “Development of Animal Models and Related Biological Materials for Research (R21)” (NOT-OD-12-054)

The Collaborative Islet Transplantation Registry (UC4) (RFA-DK-12-002)

Government-Owned Inventions; Availability for Licensing (FR Doc No: 2012-4736)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-4748)

Center For Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-5046)

National Cancer Institute; Notice of Closed Meetings (FR Doc No: 2012-4922)

Clinical Center Notice of Meeting (FR Doc No: 2012-4936)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-4907)