Volume 17.04 | Feb 1

Issue 17.04 February 1, 2016
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Viral Gene Editing System Corrects Genetic Liver Disease in Newborn Mice
For the first time, researchers have treated an animal model of a genetic disorder using a viral vector to deliver genome-editing components in which the disease-causing mutation has been corrected. [Press release from the University of Pennsylvania discussing online prepublication in Nature Biotechnology] Press Release | Abstract
Learn more about our standardized tools for cord blood banking
PUBLICATIONS (Ranked by impact factor of the journal)
Targeting Human Melanoma Neoantigens by T Cell Receptor Gene Therapy
Investigators generated T cells expressing a mutation-specific transgenic T cell receptor (TCR) to target different immunogenic mutations in cyclin-dependent kinase 4 that naturally occur in human melanoma. In a syngeneic HLA-A2–transgenic mouse model of large established tumors, they found that both mutations differed dramatically as targets for TCR-modified T cells in vivo. [J Clin Invest] Full Article

Stem Cells of the Suture Mesenchyme in Craniofacial Bone Development, Repair and Regeneration
Scientists provide the first evidence for an Axin2-expressing stem cell population with long-term self-renewing, clonal expanding and differentiating abilities during calvarial development and homeostastic maintenance. [Nat Commun] Full Article | Press Release

Induction of Mesenchymal Stem Cell Chondrogenic Differentiation and Functional Cartilage Microtissue Formation for In Vivo Cartilage Regeneration by Cartilage Extracellular Matrix-Derived Particles
Functional cartilage microtissue aggregates of bone marrow stromal cell-laden cartilage extracellular matrix-derived particles formed as time in culture increased. The microtissue aggregates were directly implanted into trochlear cartilage defects in a rat model. [Acta Biomater] Abstract

Integration of 3D Printed and Micropatterned Polycaprolactone Scaffolds for Guidance of Oriented Collagenous Tissue Formation In Vivo
Researchers developed poly(ε-caprolactone) scaffolds with mesoscale and microscale architectural cues specific to human ligament progenitor cells and assessed their ability to form aligned bone–ligament–cementum complexes in vivo. [Adv Healthc Mater] Abstract

Healthy and Diseased Corticospinal Motor Neurons Are Selectively Transduced upon Direct AAV2-2 Injection into the Motor Cortex
Researchers report that adeno-associated virus (AAV)2-2 transduces corticospinal motor neurons with high efficiency upon direct cortex injection and that transduction efficiencies are similar during presymptomatic and symptomatic stages in hSOD1G93A transgenic amyotrophic lateral sclerosis mice. [Gene Ther]
Full Article | Press Release

Oncolytic Vaccinia Virus as a Vector for Therapeutic Sodium Iodide Symporter Gene Therapy in Prostate Cancer
Scientists evaluated the therapeutic potential of vaccinia virus expressing the sodium iodide symporter (NIS) in prostate cancer models, combining oncolysis, external beam radiotherapy, and NIS-mediated radioiodide therapy. In vivo experiments were carried out in PC3 xenografts in CD1 nude mice to assess NIS expression and tumor radioiodide uptake. [Gene Ther] Full Article

Engraftment of Enteric Neural Progenitor Cells into the Injured Adult Brain
The authors propose an innovative approach that utilizes gut-derived neural tissue for cell-based therapies following focal or diffuse central nervous system injury. Using different models of brain injury in combination with either local or systemic cell delivery, they showed that transplanted enteric neuronal progenitor cells survive, proliferate, and differentiate into neuronal and glial lineages in vivo. [BMC Neurosci] Full Article

Induction of eEF2-Specific Antitumor CTL Responses In Vivo by Vaccination with eEF2-Derived 9mer-Peptides
To examine in vivo antitumor effects of eukaryotic elongation factor 2 (eEF2) peptide vaccination in a therapeutic model, mice were vaccinated four times with one each of the two eEF2 peptides at weekly intervals after implantation of eEF2-expressing leukemia cells. The vaccination with eEF2 peptides induced eEF2-specific cytotoxic T lymphocytes (CTLs) and suppressed tumor growth, and disease-free survival was significantly longer in EF180-vaccinated mice compared to control mice. [Oncol Rep] Abstract

Transplantation of CXCR4 Overexpressed Mesenchymal Stem Cells Augments Regeneration in Degenerated Intervertebral Discs
Investigators aimed to evaluate whether high expression of CXCR4 enhances the migration of mesenchymal stem cells and increases the efficiency of intervertebral disc regeneration. [DNA Cell Biol] Abstract

Watch Video MesenTrek Rise of Enriched Mouse MSC Cultures
Manufacturing of AcMNPV Baculovirus Vectors to Enable Gene Therapy Trials
The authors provide an overview of the current approaches for scaled-up amplification, concentration, purification, and formulation of AcMNPV baculoviruses, and highlights the key regulatory requirements that must be met before gene therapy clinical trials can be initiated. [Mol Ther Methods Clin Dev] Full Article

Targeted Approaches to Induce Immune Tolerance for Pompe Disease Therapy
Investigators highlight the advances in therapies as well as the improved understanding of the molecular mechanisms involved in the humoral immune response with emphasis on methods employed to overcome responses associated with enzyme and gene therapies for Pompe disease. [Mol Ther Methods Clin Dev] Full Article

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

New Cell Separation Kits to Isolate 90% Pure CD34+ Cells from Cord Blood. See the data and learn more here!
Genticel Reports Initial Results at 12 Months from Phase II Trial of HPV Immunotherapeutic Candidate, GTL001
Genticel announced initial results from the ongoing randomized, double-blind, placebo controlled Phase II clinical study of its immunotherapeutic candidate, GTL001, designed to clear HPV 16 and/or 18 infection. [Genticel] Press Release

Study Shows Different Genetic Drivers of Colorectal Cancer in Older and Younger Patients
A University of Colorado Cancer Center study showed genetic differences between colorectal cancer in young and old patients, possibly pointing toward different treatments and strategies in combating the young form of the disease. [Press release from the University of Colorado discussing research presented at the 2016 Gastrointestinal Cancers Symposium, San Francisco] Press Release

Register for Adoptive T-Cell Therapy Congress
UK Scientists Gain License to Edit Genes in Human Embryos
Scientists in London have been granted permission to edit the genomes of human embryos for research, UK fertility regulators announced. The approval by the UK Human Fertilization and Embryology Authority represents the world’s first endorsement of such research by a national regulatory authority. [Nature News] Editorial

MD Anderson Joins Nation’s Cancer Centers in Endorsement of HPV Vaccination for Cancer Prevention
In response to low national vaccination rates for the human papillomavirus (HPV), The University of Texas MD Anderson Cancer Center has joined with the 68 other National Cancer Institute (NCI)-designated cancer centers in issuing a statement calling for increased HPV vaccination for the prevention of cancer. [The University of Texas MD Anderson Cancer Center] Press Release | Statement

From our sponsor: Learn about applications of ALDHbr cells in regenerative medicine. Watch the video.
ImmunoCellular Therapeutics Enters into a Sponsored Research Agreement with the University of Maryland, Baltimore to Pursue Multiple Enhancements of Its Cancer Immunotherapy Platforms
ImmunoCellular Therapeutics, Ltd. announced it has entered into a sponsored research agreement with the University of Maryland School of Medicine, and the University of Maryland, Baltimore. The agreement includes three projects, which together have the potential to improve the efficacy of dendritic cell, T-cell, and combination immunotherapies for cancer and lead to enhancements to both of ImmunoCellular’s dendritic cell and Stem-to-T-cell platforms. [ImmunoCellular Therapeutics, Ltd.] Press Release

PCI Biotech and Ultimovacs Initiates Preclinical Research Collaboration
PCI Biotech and Ultimovacs announced that they are initiating a preclinical research collaboration. The preclinical research collaboration will evaluate technology compatibility and synergy based on in vivo studies. [PCI Biotech] Press Release

Wyss Institute Will Lead IARPA-Funded Brain Mapping Consortium
The Wyss Institute for Biologically Inspired Engineering announced a cross-institutional consortium to map the brain’s neural circuits with unprecedented fidelity. The consortium is made possible by a $21 million contract from the Intelligence Advanced Research Projects Activity (IARPA) and aims to discover the brain’s learning rules and synaptic ‘circuit design’, further helping to advance neurally-derived machine learning algorithms. [Wyss Institute] Press Release

Feds Back New Heart Patch for Infants
Bioengineers at Rice University and Texas Children’s Hospital have won a National Institutes of Health grant to develop a new generation of patches to repair the damaged hearts of infants. [Rice University] Press Release

Phase II Colorectal Cancer Clinical Trial First to Receive Support, Scientific Oversight from Van Andel Research Institute–Stand Up to Cancer Epigenetics Dream Team
The first clinical trial to move forward as part of the Van Andel Research Institute–Stand Up to Cancer Epigenetics Dream Team will target metastatic colorectal cancer, the second leading cause of cancer deaths among men and women combined in the U.S. [Van Andel Research Institute] Press Release

Clovis Oncology Initiates Immunotherapy Combination Trial
Clovis Oncology, Inc. announced that it initiated a clinical trial to evaluate a novel combination therapy of Genentech’s investigational cancer immunotherapy atezolizumab and rociletinib for the treatment of advanced EGFR-mutant non-small cell lung cancer. [Clovis Oncology, Inc.] Press Release

Cynata Receives Favorable Advice from UK Regulatory Authority
The UK Medicines and Healthcare products Regulatory Agency confirmed that Cynata’s proprietary Cymerus™ mesenchymal stem cell product, CYP-001, is considered to be suitable for use in a proposed Phase I clinical trial in patients with graft versus host disease. [Cynata Therapeutics Ltd] Press Release

TRACON Announces FDA Orphan Drug Designation for TRC105 in Soft Tissue Sarcoma
TRACON Pharmaceuticals announced that the U.S Food and Drug Administration (FDA) has granted TRC105 orphan drug designation for the treatment of patients with soft tissue sarcoma. TRC105 is a novel, clinical stage antibody to endoglin, a protein overexpressed on proliferating endothelial cells that is essential for angiogenesis. [TRACON Pharmaceuticals, Inc.] Press Release
National Cancer Institute; Notice of Closed Meetings (FR Doc. No: 2016-01696)

Center For Scientific Review; Notice of Closed Meetings (FR Doc. No: 2016-01364)
Cellular, Tissue and Gene Therapies Advisory Committee; Notice of Meeting
Food and Drug Administration (United States)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Public Health Service Guideline on Infectious Disease Issues in Xenotransplantation (FR Doc. No: 2016-01638)
NEW Advanced Cell Therapy Symposium
March 14-15, 2016
London, United Kingdom

NEW International Conference on Human & Translational Immunology
September 16-21, 2016
Rhodes, Greece

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Position – Hematology/Oncology (Penn State College of Medicine)

NEW Group Leader – Cell Therapy (Miltenyi Biotec)

NEW Director of Manufacturing – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Research Associate – Rheumatology (Newcastle University)

Professorship – Transfusion Medicine and Cell Therapeutics (Ludwig-Maximilians-Universität München)

Postdoctoral Fellow/Research Associate – Cancer Therapy (Memorial Sloan Kettering Cancer Center)

Translational Research Scientist – Hematology/Oncology (University of Chicago)

Postdoctoral Position – Cancer Immunotherapy (German Cancer Research Center)

Staff Scientist – Cell Therapy Process Development (Fred Hutchinson Cancer Research Center)

Postdoctoral Position – CAR T Cell Engineering & Cancer Immunotherapy (University Hospital Würzburg)

Postdoctoral Research Fellow – Cell Cycle Control and Tumorigenesis (Fred Hutchinson Cancer Research Center)

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