Cell Therapy News Volume 15.22 | Jun 9 2014

A Single Delivery of an Adeno-Associated Viral Construct to Transfer the CASQ2 Gene to Knock-In Mice Affected by Catecholaminergic Polymorphic Ventricular Tachycardia Is Able to Cure the Disease from Birth to Advanced Age
Scientists investigated the efficacy and durability of viral gene transfer of Calsequestrin 2 (CASQ2) wild type gene in a catecholaminergic polymorphic ventricular tachycardia knock-in mice model carrying the CASQ^2R33Q/R33Q mutation. [Circulation] Abstract

Protective Effects of Fc-Fused PD-L1 on Two Different Animal Models of Colitis
Researchers investigated the effects of programmed death-ligand 1 (PD-L1)-Fc treatment on intestinal inflammation using two murine models of inflammatory colitis induced by dextran sulfate sodium and T-cell transfer. [Gut] Abstract

Collecting Duct-Derived Cells Display Mesenchymal Stem Cell Properties and Retain Selective In Vitro and In Vivo Epithelial Capacity
After extensive passage, kidney mesenchymal stem cell-like cells selectively integrated into the aquaporin 2-positive medullary collecting duct when microinjected into the kidneys of neonatal mice. [J Am Soc Nephrol] Abstract

In Vivo Targeted Delivery of CD40 shRNA to Mouse Intestinal Dendritic Cells by Oral Administration of Recombinant Sacchromyces cerevisiae
Scientists demonstrated that recombinant yeast S. cerevisiae efficiently delivered the short hairpin RNA of immune-associated gene into mouse intestinal dendritic cells via oral administration. [Gene Ther] Full Article

Preclinical Evaluation of IL2-Based Immunocytokines Supports Their Use in Combination with Dacarbazine, Paclitaxel and TNF-Based Immunotherapy
Investigators used the K1735M2 immunocompetent syngeneic model of murine melanoma to study the therapeutic activity of F8-IL2, an immunocytokine based on the F8 antibody in diabody format, fused to human interleukin-2 (IL2). [Cancer Immunol Immunother] Abstract

Undifferentiated and Differentiated Adipose-Derived Stem Cells Improve Nerve Regeneration in a Rat Model of Facial Nerve Defect
In an in vivo study, a silicone conduit containing undifferentiated adipose-derived stem cells (ASCs), differentiated ASCs or Schwann cells were transplanted, embedded in a collagen gel and the efficacy of repair of a 7 mm-gap in the rat facial nerve examined. [J Tissue Eng Regen Med] Abstract

Knockdown of Toll-Like Receptor 4 Inhibits Human NSCLC Cancer Cell Growth and Inflammatory Cytokine Secretion In Vitro and In Vivo
The authors sought to characterize the expression of toll-like receptor 4 (TLR4) in patients with non-small cell lung cancer (NSCLC) and to investigate the biological roles of TLR4 in lung metastasis, cell invasion and survival. They showed that downregulation of TLR4 expression using an RNA silencing approach in A549 tumor cells significantly suppressed cell proliferation, cell migration and cell invasion, and induced tumor apoptosis in vitro, and suppressed tumor growth in vivo. [Int J Oncol] Abstract

Mesenchymal Stromal Cell Therapy Is Associated with Increased Adenovirus-Associated but Not Cytomegalovirus-Associated Mortality in Children with Severe Acute Graft-versus-Host Disease
Investigators evaluated 56 patients with grade II-IV acute graft-versus-host disease who responded to steroids or were steroid refractory receiving either mesenchymal stromal cells (MSCs) or other second-line therapy. Although the overall incidence of cytomegalovirus, Epstein-Barr virus, and human adenovirus (HAdV) infections was not significantly increased, HAdV infection was associated with decreased survival in children treated with MSCs. [Stem Cells Transl Med] Abstract

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Applications of TALENs and CRISPR/Cas9 in Human Cells and Their Potentials for Gene Therapy
The authors review the working mechanism of TALEN and CRISPR/Cas9, their effectiveness and specificity in human cells, and current methods to enhance efficiency and reduce off-target effects. [Mol Biotechnol] Abstract

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

uniQure Announces Analysis of Six-Year Follow-Up Data for Glybera^®
uniQure N.V. announced the full analysis of the six-year follow-up data for patients treated once with Glybera^® (alipogene tiparvovec), uniQure’s gene therapy product for the treatment of lipoprotein lipase deficiency. [Press release from uniQure N.V. discussing research presented at the 82^nd Annual Congress of the European Atherosclerosis Society (EAS), Madrid] Press Release

Long-Term Results Encouraging for Combination Immunotherapy for Advanced Melanoma
The first long-term follow-up results from a Phase Ib immunotherapy trial combining drugs for advanced melanoma patients has shown encouraging results – long-lasting with high survival rates – researchers report. [Press release from Yale University discussing research presented at the 50^th Annual Meeting of the American Society of Clinical Oncology (ASCO), Chicago] Press Release

Advaxis’s ADXS-cHER2 Immunotherapy Continues to Demonstrate Significantly Prolonged Survival in Canine Osteosarcoma
Advaxis, Inc. presented updated data from the ongoing ADXS-cHER2 study in canine osteosarcoma. The preliminary findings of the Phase I clinical trial in dogs with osteosarcoma suggest that ADXS-cHER2 is safe and well tolerated at doses up to 3 x 10⁹ CFU with no evidence of cardiac, hematological, or other systemic toxicities. [Press release from GlobeNewswire, Inc. discussing research presented at the 2014 American College of Veterinary Internal Medicine (ACVIM) Forum, Nashville] Press Release

Last Remaining Support for Controversial Stem-Cell Papers Collapses
The retraction of two controversial papers that promised a simple way to create embryonic-like stem cells seems to be imminent after the lead author unexpectedly gave her full consent. Haruko Obokata, of the RIKEN Center for Developmental Biology in Kobe, Japan, had been the last obstacle to the retraction of both papers. [Nature News] Editorial

Output Drops at World’s Largest Open-Access Journal
The number of papers published by the world’s largest open-access journal, PLoS One, has plummeted over the past few months after rising fairly steadily for years, notes a scholarly publishing blogger. Phil Davis suggests the closely watched PLoS One may have become a less attractive option for scientists as its impact factor has fallen and other open-access publishers have come on the scene. [ScienceInsider] Editorial

NSF Counsel Lashes Out at Scientists Asking about Protections for Rotators
The National Science Foundation’s (NSF’s) top lawyer has rebuked a group of U.S. scientists who asked for an explanation of its policies governing temporary workers. The response appears to have widened a rift between that community and NSF over a program designed to keep the agency on the cutting edge of research. [ScienceInsider] Editorial

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Cellectis and Accelera (Nerviano Medical Sciences Group) Sign an Agreement to Complete Preclinical Studies of Cellectis’ Lead Product Candidate UCART19
Cellectis and Accelera signed an agreement to complete the preclinical studies of Cellectis’ advanced product candidate, UCART19. Engineered allogeneic CD19 T-cells currently stand out as a real therapeutic innovation for treating B-cell leukemias and lymphomas. [Cellectis] Press Release

arGEN-X Enters Long-Term Strategic Alliance with Shire Pharmaceuticals in Therapeutic Antibodies
arGEN-X announced it has entered into a long-term strategic alliance with Shire Pharmaceuticals. Under the agreement, arGEN-X will bring its entire suite of human antibody discovery technologies to a partnership focused on multiple targets aligned with Shire’s therapeutic focus. [arGEN-X B.V.] Press Release

REGENX Biosciences and Voyager Therapeutics Announce License Agreement
REGENX Biosciences, LLC and Voyager Therapeutics announced that they have entered into a license agreement for use of REGENX’s proprietary NAV^® vectors for the development and commercialization of gene therapies to treat amyotrophic lateral sclerosis, Friedreich’s ataxia and Huntington’s disease. [Voyager Therapeutics]
Press Release

Conrad T. Prebys Gives $25 Million to Salk Institute to Support Scientific Research
The Salk Institute for Biological Studies has received a $25 million gift from San Diego philanthropist and former Salk trustee Conrad T. Prebys to support cutting-edge biological research on a wide range of diseases. [The Salk Institute for Biological Studies] Press Release

UCLA Stem Cell Researcher Receives $5.1 Million Award from State Agency
California’s state stem cell agency has given UCLA’s Dr. John Chute a Research Leadership Award worth $5.1 million to support his research aimed at creating new stem cell therapies for use in medical practice. [UCLA]
Press Release

Medigene AG: Trianta Receives BMBF (Federal Ministry of Education and Research) Grant for TABs Immunotherapy as Part of the “m4 Leading-Edge Cluster Initiative”
Medigene AG announced its subsidiary Trianta Immunotherapies GmbH will receive public funding for the development of its immunotherapy platform TABs (T-cell-specific antibodies) for the treatment of various types of cancer and autoimmune diseases. [Medigene AG] Press Release

Medgenics Announces First Patient Implanted in Phase I/II Clinical Trial of MDGN-201 (EPODURE)
Medgenics, Inc., the developer of a novel platform technology for the sustained production and delivery of therapeutic proteins in patients using ex-vivo gene therapy and their own tissue for the treatment of rare and orphan diseases, announced that the first patient has been enrolled in its Phase I/II clinical trial of MDGN-201. [Medgenics, Inc.] Press Release

Enzo Biochem Awarded U.S. Patent on More Efficient Vector for Use in Gene Therapy
Enzo Biochem Inc. announced that it has been assigned U.S. Patent Office Patent No. 8,742,090, related to the construct of an artificial vector capable of greater efficiency, in general, in targeting specific cells in the application of gene therapy and gene transfer technology. [Business Wire] Press Release

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Prescription Drug Advertisements (FR Doc. No: 2014-13015)

Determination of Regulatory Review Period for Purposes of Patent Extension; XIAFLEX (FR Doc. No: 2014-12808)

NEW World Alliance Forum in San Francisco – The Impact of Regenerative Medicine 2014
November 6-7, 2014
San Francisco, United States

NEW The World Stem Cell Summit 2014
December 3-5, 2014
San Antonio, United States

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

Postdoctoral Fellow – Prostate Cancer Research (Henry Ford Hospital)

Cell Biologists – T Cell-Based Therapies (Adaptimmune)

Postdoctoral/Clinical Pharmacist – Hematopoiesis and Laboratory for Cell Therapy (Sanquin)

Postdoctoral Position – Neurotoxicity and Epigenetics (Thomas Jefferson University)

Research/Review Scientist – Cell Therapy and Regenerative Medicine (Center for Biologics Evaluation and Research)

Postdoctoral Position – Cell Death and Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Position – Role of Calreticulin in TGF-Beta Signaling in the Kidney Proximal Tubule under Diabetic Conditions (University of Alabama)

Postdoctoral Fellow – Transgene Immunity in AAV Vector-Based Antiviral and/or Gene Therapeutics (University of Massachusetts)

Postdoctoral Fellow – Neurophysiology of Addiction (Medical University of South Carolina)

Principal Investigator – Molecular Mechanisms of Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Associate – Hematopoietic Differentiation (Northwestern University Feinberg School of Medicine)

Developmental-Stem Cell Biologist (Mayo Clinic – Rochester)

Postdoctoral Fellowship – Hematology, Immunology, Cancer Therapeutics (Nationwide Children’s Hospital)

Scientist – Immunology/Cell Separation (STEMCELL Technologies Inc.)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

Research Technologist – Pluripotent Stem Cells (STEMCELL Technologies Inc.)

Research Technologist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

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