Volume 16.44 | Nov 30

Issue 16.44 November 30, 2015
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Normalizing the Levels of MeCP2 in a Mouse Model of MECP2 Duplication Syndrome Restores Neurological Function
Using both state-of-the-art genetic techniques and a small molecule that can target specific genetic material, researchers have shown that it is possible to reverse the terrible effects of the duplicated gene MECP2 (methyl-CpG-binding protein). [Press release from Baylor College of Medicine discussing online prepublication in Nature] Press Release | Abstract
New Cell Separation Kits to Isolate 90% Pure CD34+ Cells from Cord Blood. See the data and learn more here!
PUBLICATIONS (Ranked by impact factor of the journal)
The Noncoding RNAs SNORD50A and SNORD50B Bind K-Ras and Are Recurrently Deleted in Human Cancer
To explore their role in cancer, investigators compared 5,473 tumor-normal genome pairs to identify small nucleolar RNAs (snoRNAs) with frequent copy number loss. The SNORD50A-SNORD50B snoRNA locus was deleted in 10–40% of 12 common cancers, where its loss was associated with reduced survival. [Nat Genet] Abstract | Press Release

Trastuzumab Emtansine (T-DM1) Renders HER2+ Breast Cancer Highly Susceptible to CTLA-4/PD-1 Blockade
Scientists showed that drug resistance can be overcome with trastuzumab emtansine, an antibody-drug conjugate that combines the human epidermal growth factor receptor 2 (HER2)-targeting ability of trastuzumab with a cytotoxic drug, which the antibody delivers directly to the tumor. [Sci Transl Med] Abstract | Press Release

Type 1 Diabetes Immunotherapy Using Polyclonal Regulatory T Cells
Researchers report a Phase I trial of adoptive regulatory T cells (Tregs) immunotherapy to repair or replace Tregs in type 1 diabetics. The ex vivo–expanded polyclonal Tregs were long-lived after transfer and retained a broad Tregs phenotype long-term. [Sci Transl Med] Abstract | Press Release

PDL1 Regulation by p53 via miR-34
Investigators showed that PDL1 is regulated by p53 via miR-34. Therapeutic delivery of miR-34a–loaded liposomes promoted tumor-infiltrating lymphocytes and reduced CD8+PD1+ cells in vivo. [J Natl Cancer Inst] Abstract | Press Release

Engraftment of Human Induced Pluripotent Stem Cell-Derived Hepatocytes in Immunocompetent Mice via 3D Co-Aggregation and Encapsulation
Scientists demonstrated successful engraftment of human induced pluripotent stem cell-derived hepatocyte-like cells in immunocompetent mice by pre-engineering 3D cell co-aggregates with stromal cells followed by encapsulation in recently developed biocompatible hydrogel capsules. [Sci Rep] Full Article

Bone Marrow Mesenchymal Stem Cell Aggregate: An Optimal Cell Therapy for Full-Layer Cutaneous Wound Vascularization and Regeneration
Cell-aggregates constructed with green fluorescent protein-expressing rat bone marrow mesenchymal stem cells were applied to rat acute full-layer cutaneous wound model to confirm its pro-regeneration ability and compare its regenerative efficacy with the currently thriving subcutaneous and intravenous stem cell administration strategy, with a view to sensing the advantages, disadvantages and the mechanism behind. [Sci Rep] Full Article

In Situ Vascular Regeneration Using Substance P-Immobilized Poly(L-Lactide-Co-ε-Caprolactone) Scaffolds: Stem Cell Recruitment, Angiogenesis, and Tissue Regeneration
Investigators fabricated scaffolds by mixing appropriate proportions of linear poly (l-lactide-co-ε-caprolactone) (PLCL) and substance P-immobilized PLCL. The biocompatibility and biological performance of the scaffolds were evaluated by in vivo experiments involving subcutaneous scaffold implantations in Sprague-Dawley rats for up to 28 days followed by histological and immunohistochemical studies. [Eur Cell Mater] Abstract | Full Article

Arterial Decellularized Scaffolds Produced Using an Innovative Automatic System
Researchers developed an innovative system for the automatic decellularization of blood vessels, used to process pig arteries. Decellularized scaffolds were seeded on the inner layer with human endothelial cells and implanted as iliac artery replacement in four pharmacologically immune-compromised pigs. [Cells Tissues Organs] Abstract

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Humanized Mouse Models for Transplant Immunology
The authors highlight recent advances in the development of humanized mice and their use as preclinical models for the study of human allograft responses. [Am J Transplant] Abstract

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

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Combination of Bevacizumab and Lomustine with First Recurrence of Glioblastoma Prolongs PFS but Not OS
Results of EORTC trial 26101 showed that bevacizumab treatment in patients with progressive glioblastoma, despite prolonged progression-free survival (PFS), does not confer a survival advantage. [Press release from the European Organisation for Research and Treatment of Cancer (EORTC) discussing research presented at the 20th Annual Scientific Meeting of the Society for Neuro-Oncology (SNO), San Antonio] Press Release

Merck Announces Initial Results for KEYTRUDA® (Pembrolizumab) with Novel Immunotherapy Combinations from Three Investigational Studies Presented
Merck announced findings from three studies investigating the use of KEYTRUDA®, the company’s anti-PD-1 therapy, in combination with three other immunotherapies – epacadostat, IMLYGICTM, and ipilimumab – in patients with advanced melanoma. [Press release from Merck & Co., Inc. discussing research presented at the 12th International Congress of the Society for Melanoma Research (SMR), San Francisco] Press Release

Cell Therapy Manufacturing & Gene Therapy Congress
U.K. Science Spared the Budget Ax and Bolstered against Inflation
Funding for science in the United Kingdom will remain constant in real terms at £4.7 billion per year until 2021, according to a government spending review. However, some noted that many details remain unclear. [ScienceInsider] Editorial

Brazilian Courts Tussle over Unproven Cancer Treatment
A court in the Brazilian state of São Paulo has cut off distribution of a compound that is hailed by some as a miracle cancer cure — even though it has never been formally tested in humans. [Nature News] Editorial

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New EU Project Designed to Link Diagnosis and Treatment of Diseases over the Long Term
An international team from four EU countries plans to use an innovative concept to improve the use of companion diagnostics in disease and develop new approaches to therapy in the long term. [Johannes Gutenberg-Universität Mainz] Press Release

New Agreement to Open the Door to Next Generation Cell Therapies
A new collaboration agreement has been announced by Macquarie University and clinical-stage regenerative medicine company Regeneus Ltd., to commercialize a breakthrough cell selection technology that for the first time allows high potency secreting stem cells to be identified and selected for the manufacture of next generation cell therapies. [Regeneus Ltd.] Press Release

CPRIT Awards Faculty $19.6 Million for Research in Cervical, Breast, Lung, Colon, and Pediatric Cancers, and Cancer Biology
The Cancer Prevention and Research Institute of Texas (CPRIT) has awarded UT Southwestern Medical Center researchers more than $19.6 million in research grants to improve preventive, diagnostic, and therapeutic services relating to cervical, breast, lung, colon, and pediatric cancers, as well as to improve scientific understanding of cancer biology. [UT Southwestern Medical Center] Press Release

MassGeneral Hospital for Children Receives Grant to Pursue Novel Antibacterial Strategy
MassGeneral Hospital for Children has been named a Grand Challenges Explorations winner through an initiative funded by the Bill & Melinda Gates Foundation. Researchers will pursue an innovative global health and development research project, titled “Disarming Type III Secretion System of Enteric Pathogens.” [MassGeneral Hospital (EurekAlert!)] Press Release

Pembrolizumab in Advanced Melanoma: Added Benefit for Certain Patients
The German Institute for Quality and Efficiency in Health Care examined in a dossier assessment whether pembrolizumab offers an added benefit over the appropriate comparator therapy. According to the findings, an added benefit can be derived for different patient groups. [German Institute for Quality and Efficiency in Health Care] Press Release

New Patent Paves Way for Breast Cancer Prevention
George Mason University researchers have patented a new breast cancer treatment that uses a common malaria drug to stop cancer in its beginning stages. Chloroquine, a drug commonly given to prevent or treat malaria, targets ductal carcinoma in situ, the most common type of pre-invasive breast cancer. [George Mason University] Press Release

FDA Approves Portrazza to Treat Advanced Squamous Non-Small Cell Lung Cancer
The U.S. Food and Drug Administration approved Portrazza (necitumumab) in combination with two forms of chemotherapy to treat patients with advanced squamous non-small cell lung cancer who have not previously received medication specifically for treating their advanced lung cancer. [U.S. Food and Drug Administration] Press Release

Adaptimmune Initiates Phase I/II Trial Evaluating Its Affinity Enhanced T-Cell Therapy Targeting
Adaptimmune Therapeutics plc announced that it has initiated a study of its affinity enhanced T-cell therapy targeting the NY-ESO-1 cancer antigen in patients with Stage IIIb or Stage IV non-small cell lung cancer. [Adaptimmune Therapeutics plc] Press Release
Final Human Immunodeficiency Virus (HIV) Organ Policy Equity (HOPE) Act Safeguards and Research Criteria for Transplantation of Organs Infected with HIV (FR Doc. No: 2015-30172)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-29940)
Vaccines and Related Biological Products Advisory Committee; Notice of Meeting
Food and Drug Administration (United States)

Agency Information Collection Activities; Announcement of Office of Management and Budget Approval; Guidance for Industry on Formal Meetings between the Food and Drug Administration and Biosimilar Biological Product Sponsors or Applicants (FR Doc. No: 2015-27558)
NEW International Society for Cellular Therapy (ISCT) 2016
May 25-28, 2016
Singapore, Singapore

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Position – Neurosensory Stem Cell Biology and Regenerative Therapy of the Inner Ear (Universitätsklinikum Tübingen)

Research Technician – Preclinical and Translational Research (Qu Biologics Inc.)

Investigator – Cancer Biology (Wake Forest Baptist School of Medicine)

Staff Scientist – Immunology and Clinical Research (Fred Hutchinson Cancer Research Center)

Postdoctoral Position – Scaffold Design & Drug/Gene Delivery for Neural Tissue Engineering (Nanyang Technological University)

Postdoctoral Position – Stem Cell Engineering Research (Nanyang Technological University)

Postdoctoral Position – hPSC-Derived Cardiomyocyte Grafts in Injured Hearts (University Health Network)

Group Leader – Translational Research (Cancer Research Center of Toulouse)

Group Leader Position – Cancer Research (Oslo University Hospital)

Research Technician – TCR Gene Therapy (Fred Hutchinson Cancer Research Center)

Research Technician – Stem Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Assistant/Associate/Full Professor – Biomedical Engineering (Worcester Polytechnic Institute)

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