Cell Therapy News Volume 14.06 | Feb 18 2013

May 12, 2022

Issue 14.06 February 18, 2013

TOP STORY

Synthetic Circuit Allows Dialing Gene Expression Up or Down in Human Cells
Scientists who built a synthetic gene circuit that allowed for the precise tuning of a gene’s expression in yeast have now refined this new research tool to work in human cells. [Press release from The University of Texas MD Anderson Cancer Center discussing online prepublication in Nature Communications] Press Release | Abstract

PUBLICATIONS (Ranked by impact factor of the journal)

NANOG-Dependent Function of TET1 and TET2 in Establishment of Pluripotency
Whereas the genomic occupancy of NANOG has been extensively investigated, comparatively little is known about NANOG-associated proteins and their contribution to the NANOG-mediated reprogramming process. Using enhanced purification techniques and a stringent computational algorithm, researchers identified 27 high-confidence protein interaction partners of NANOG in mouse embryonic stem cells. They confirmed physical association of NANOG with ten-eleven translocation 1 (TET1), and demonstrated that TET1, in synergy with NANOG, enhances the efficiency of reprogramming. [Nature] Abstract | Press Release

Novel Interferon-Based Pre-Transplantation Conditioning in the Treatment of a Congenital Metabolic Disorder
The authors showed that type I IFN preconditioning, without irradiation or DNA alkylating agents, significantly enhanced the hematopoietic stem cell engraftment efficiency in wild type recipient mice. [Blood] Abstract

Astrocyte Pathology and the Absence of Non-Cell Autonomy in an Induced Pluripotent Stem Cell Model of TDP-43 Proteinopathy
Investigators generated functional astroglia from human induced pluripotent stem cells carrying an amyotrophic lateral sclerosis-causing transactive response DNA-binding protein (TDP-43) mutation and showed that mutant astrocytes exhibit increased levels of TDP-43, subcellular mislocalization of TDP-43, and decreased cell survival. [Proc Natl Acad Sci USA] Abstract | Full Article | Press Release

Autologous Adipose Tissue-Derived Stem Cells Treatment Demonstrated Favorable and Sustainable Therapeutic Effect for Crohn’s Fistula
In a Phase I clinical trial, adipose tissue-derived stem cells (ASCs) demonstrated their safety and therapeutic potential for healing fistulae associated with Crohn’s disease. This study was carried out to evaluate the efficacy and safety of ASCs in patients with Crohn’s fistulae. In this Phase II study, forty-three patients were treated with ASCs. [Stem Cells] Abstract | Full Article

Enhancing Chemotherapy Response with Sustained EphA2 Silencing Using Multistage Vector Delivery
Researchers evaluated therapeutic efficacy of multistage vector-loaded EphA2 small inhibitory RNA with murine orthotopic models of metastatic ovarian cancers as a first step towards development of a new class of nanotherapeutics for the treatment of ovarian cancer. [Clin Cancer Res] Abstract | Press Release

Lentiviral Delivery of RNAi for In Vivo Lineage-Specific Modulation of Gene Expression in Mouse Lung Macrophages
Researchers report the use of lentiviral vectors for long-term in vivo delivery of RNA interference (RNAi) selectively to resident alveolar macrophages, key immune effector cells in the lung. They demonstrated the therapeutic potential of this approach by RNAi-based downregulation of p65, a component of the pro-inflammatory transcriptional regulator, nuclear factor κB and a key participant in lung disease pathogenesis. [Mol Ther] Abstract

Site-Specific Gene Delivery to Stented Arteries Using Magnetically Guided Zinc Oleate-Based Nanoparticles Loaded with Adenoviral Vectors
The authors hypothesized that novel zinc oleate-based magnetic nanoparticles loaded with adenovirus (Ad) would enable effective arterial cell transduction by shifting vector processing to an alternative pathway, protect Ad from inactivation by neutralizing factors, and allow site-specific gene transfer to arteries treated with stent angioplasty using a two-source magnetic guidance strategy. [FASEB J] Abstract

Recovery of Behavioral Symptoms in Hemi-Parkinsonian Rhesus Monkeys through Combined Gene and Stem Cell Therapy
Neuronal-primed adipose mesenchymal stem cells derived from rhesus monkey combined with adenovirus containing Neurturin and tyrosine hydroxylase were implanted into the striatum and substantia nigra of methyl-4-phenyl-1, 2, 3, 6-tetrahydropyridine-lesioned hemi-parkinsonian rhesus monkeys. [Cytotherapy] Abstract

Adipose Stromal Cells Primed with Hypoxia and Inflammation Enhance Cardiomyocyte Proliferation Rate In Vitro through STAT3 and Erk1/2
Researchers demonstrated that adipose derived stromal cells enhance the proliferation rate of rat neonatal cardiomyocytes and adult HL-1 cardiomyocytes in a paracrine fashion. [J Transl Med] Abstract | Full Article

Neural Progenitors Derived from Human Induced Pluripotent Stem Cells Survive and Differentiate Upon Transplantation into a Rat Model of Amyotrophic Lateral Sclerosis
Investigators analyzed the fate of human induced pluripotent stem cell (iPSC)-derived neural progenitors transplanted into the spinal cord of wild-type and transgenic rats carrying a human mutated SOD1(G93A) gene. The aim was to follow survival and differentiation of human neural progenitors until day 60 post-transplantation in two different in vivo environments, one being amyotrophic lateral sclerosis-like. iPSC-derived neural progenitors efficiently engrafted in the adult spinal cord and survived at high numbers. [Stem Cells Transl Med]
Abstract | Press Release

REVIEWS

The Endogenous Regenerative Capacity of the Damaged Newborn Brain: Boosting Neurogenesis with Mesenchymal Stem Cell Treatment
Researchers discuss the endogenous capacity of the neonatal brain to regenerate after a cerebral ischemic insult. Furthermore, they consider the potential to use stem cell transplantation as a means to boost endogenous neurogenesis and restore brain function. [J Cereb Blood Flow Metab] Abstract

Targeted Therapy in Renal Cell Carcinoma: Moving from Molecular Agents to Specific Immunotherapy
Non-specific immunotherapy has been for a long time a standard treatment option for patients with metastatic renal cell carcinoma but was redeemed by specific targeted molecular therapies, namely the VEGF and mTOR inhibitors. Recent steps in the development of immunotherapy strategies are discussed in this review with a special focus on peptide vaccination which aims at a tumor targeting by specific T lymphocytes. [World J Urol] Full Article

SCIENCE NEWS

StemCells, Inc. Announces First Patient Cohort Completes Spinal Cord Injury Trial – Gains in Sensory Function Persist 12 Months after Stem Cell Transplant
StemCells, Inc. announced that the twelve-month data from the first patient cohort in the Company’s Phase I/II clinical trial of its proprietary HuCNS-SC® product candidate (purified human neural stem cells) for chronic spinal cord injury continued to demonstrate a favorable safety profile, and showed that the considerable gains in sensory function observed in two of the three patients at the six-month assessment have persisted. The third patient remains stable. [Press release from StemCells, Inc. discussing research presented at the 15^th Annual BIO CEO & Investor Conference, New York] Press Release

Queen’s Study Aims to Use Stem Cells to Help Save Sight of Diabetes Sufferers
Scientists at Queen’s University Belfast are hoping to develop a novel approach that could save the sight of millions of diabetes sufferers using adult stem cells. The novel REDDSTAR study (Repair of Diabetic Damage by Stromal Cell Administration) will isolate stem cells from donors, expand them in a laboratory setting and re-deliver them to a patient where they help to repair the blood vessels in the eye. [Queen’s University Belfast] Press Release

POLICY

Universities See Progress in New Rules for Managing U.S. Research
U.S. university administrators are poring over hundreds of pages of proposed changes to the rules governing how the federal government manages the money it spends on academic research. [White House Office of Management and Budget, United States] Press Release

Europe Scales Back Research Plans
The ambitious vision for Europe’s next research program dimmed when heads of the 27 member states of the European Union agreed to slash the overall budget for 2014-20, at the end of a long and acrimonious meeting. The deal scales back the budget proposed by the European Commission for its Horizon 2020 research program by about 13%, to €69.24 billion, and means that the first year of the new program will have a smaller budget than the last year of the previous one. [European Commission, European Union] Press Release

BUSINESS

JDRF and California Institute for Regenerative Medicine Increase Funding of ViaCyte
JDRF and the California Institute for Regenerative Medicine (CIRM) announced that they are providing additional funding for the development of a novel stem cell therapy by San Diego-based ViaCyte, Inc.; JDRF and CIRM will each contribute $3 million to further advance the project. [PR Newswire Association LLC] Press Release

STEMSOFT and The Canadian Blood and Marrow Transplant Group Announce Partnership
The Canadian Blood and Marrow Transplant Group is pleased to announce a partnership with STEMSOFT Software Inc. (STEMSOFT) that will result in a national registry of patients who have undergone blood and marrow transplant in Canada. [STEMSOFT Software Inc.] Press Release

Third Rock Ventures Launches Jounce Therapeutics with $47 Million Series A to Transform Cancer Treatment
Third Rock Ventures, LLC announced the formation of Jounce Therapeutics, Inc. with a $47 million Series A financing of the company. Jounce is focused on the discovery and development of first-in-class cancer immunotherapies designed to harness the patient’s immune system to seek out and attack cancerous cells and tumors. [Jounce Therapeutics, Inc.] Press Release

USC Launches Regenerative Medicine and Stem Cell Research Initiative
A new University of Southern California (USC) initiative has been introduced to support collaborative research in regenerative medicine and stem cell biology across the university, facilitating the critical move from basic research into clinical applications. [University of Southern California] Press Release

ACT’s Clinical Partner Receives FDA Approval to Initiate Clinical Trial Using the Company’s hESC-Derived Cells to Treat Severe Myopia
Advanced Cell Technology, Inc.’s (ACT) clinical partner, the University of California, Los Angeles, has received approval of its Investigator Investigational New Drug Application with the US Food and Drug Administration (FDA) to initiate a Phase I/II study using ACT’s retinal pigment epithelial cells derived from human embryonic stem cells (hESCs) to treat myopic macular degeneration, commonly known as nearsightedness. [Advanced Cell Technology, Inc.] Press Release

Lentigen Receives U.S. Orphan Drug Designation for Novel Gene Therapy for Glioblastoma Multiforme
Lentigen Corporation announced that that the U.S. Food and Drug Administration has granted orphan drug status to P140K methylguanine methyltransferase transduced human CD34 cells (product name: LG631-CD34) for bone marrow protection in the treatment of glioblastoma multiforme. [PR Newswire Association LLC]
Press Release

New Stem Cell Company Opens Lab at UConn Health Center
A new stem cell company, ImStem Biotechnology Inc., is the latest research venture to join the University of Connecticut’s (UConn) Technology Incubation Program. ImStem will utilize and commercialize the technologies developed by Dr. Ren He Xu, director of the UConn Stem Cell Core, and his post-doc Dr. Xiaofang Wang. [University of Connecticut] Press Release

International Stem Cell Expert Joins CERA
The Centre for Eye Research Australia (CERA) announced that one of the world’s most exciting, up-and-coming stem cell researchers has returned to Australia to join the Neuroregeneration Unit. Dr. Kathryn Davidson is a stem cell scientist, with an interest in how stem cells communicate and how this influences the way they behave and specialize. [Centre for Eye Research Australia] Press Release

NIH

NIH Urged to Consider Making All Applications New
With researchers facing ever-stiffer competition for scarce research dollars, advisers to the National Institutes of Health (NIH) are urging the agency to dust off an old idea for improving its peer review process. Instead of allowing researchers one more shot if a proposal is rejected, NIH would give them unlimited chances to resubmit, but consider all applications to be new. Press Release

Improvement of Animal Models for Stem Cell-Based Regenerative Medicine (R24)(PAR-13-113)

Improvement of Animal Models for Stem Cell-Based Regenerative Medicine (R01)(PAR-13-114)

Notice of NIAMS’ Participation in PAR-13-114 “Improvement of Animal Models for Stem Cell-Based Regenerative Medicine (R01)” (NOT-AR-13-013)

Improvement of Animal Models for Stem Cell-Based Regenerative Medicine (R21)(PAR-13-115)

Notice of NIAMS’ Participation in PAR-13-115 “Improvement of Animal Models for Stem Cell-Based Regenerative Medicine (R21)” (NOT-AR-13-014)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc No: 2013-03238)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc No: 2013-03639)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-03243)