Volume 16.31 | Aug 17

Issue 16.31 August 17, 2015
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Engineering a Permanent Solution to Genetic Diseases
New research demonstrates a new technology advancing the field of genome engineering. The method significantly improves the ability of scientists to target specific faulty genes and then “edit” them, replacing the damaged genetic code with healthy DNA. [Press release from the University of Alberta discussing online publication in Nature Methods] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Paclitaxel Tumor Priming Promotes Delivery and Transfection of Intravenous Lipid-siRNA in Pancreatic Tumors
Investigators evaluated whether tumor priming is functional for systemically delivered small interfering RNA (siRNA) via intravenous injection, which would subject siRNA to several additional delivery barriers and elimination processes. [J Control Release] Abstract

Long-Term Engraftment of Myogenic Progenitors from Adipose-Derived Stem Cells and Muscle Regeneration in Dystrophic Mice
Scientists examined the in vivo myogenic potential of progenitors differentiated from adipose-derived stem cells (ADSCs) using forskolin, basic fibroblast growth factor, the glycogen synthase kinase 3β inhibitor 6-bromoindirubin-3′-oxime as well as the supernatant of ADSC cultures. [Hum Mol Genet] Abstract

Antibacterial and Conductive Injectable Hydrogels Based on Quaternized Chitosan-graft-Polyaniline/Oxidized Dextran for Tissue Engineering
Researchers developed a new series of in situ forming antibacterial conductive degradable hydrogels using quaternized chitosan grafted polyaniline with oxidized dextran as crosslinker. [Acta Biomater] Abstract

UPRT, a Suicide-Gene Therapy Candidate in Higher Eukaryotes, Is Required for Drosophila Larval Growth and Normal Adult Lifespan
Scientists found that the Drosophila uracil phosphoribosyltransferase (UPRT) homolog, krishah, codes for an enzyme that is required for larval growth, pre-pupal/pupal viability and long-term adult lifespan. Their findings suggest that UPRT from all higher eukaryotes is likely enzymatically active in vivo and challenges the previous notion that the enzyme is non-essential in higher eukaryotes and cautions against targeting the enzyme for therapeutic purposes. [Sci Rep] Full Article

Engineered Cytotoxic T Lymphocytes with AFP-Specific TCR Gene for Adoptive Immunotherapy in Hepatocellular Carcinoma
Autologous dendritic cells pulsed with AFP158-166 peptides were used to stimulate alpha-fetoprotein (AFP)-specific cytotoxic T lymphocytes (CTLs). T cell receptor (TCR) α/β chain genes of AFP-specific CTLs were cloned and linked by 2A peptide to form full-length TCR coding sequence synthesized into a lentiviral vector. [Tumor Biol] Abstract

Management of Myelodysplastic Syndromes Relapsing after Allogeneic Hematopoietic Stem-Cell Transplantation: A Study by the SFGM-TC
To find out prognostic factors and to investigate different therapeutic approaches, investigators report on 147 consecutive patients who relapsed after allogeneic hematopoietic stem cell transplantation for myelodysplastic syndrome. [Biol Blood Marrow Transplant] Abstract

Effects of Human Amnion-Derived Mesenchymal Stromal Cell Transplantation in Rats with Radiation Proctitis
Therapeutic irradiation for intra-pelvic cancer often causes radiation proctitis; however, there is currently no effective treatment. Researchers investigated the effect of transplantation of human amnion-derived mesenchymal stromal cells in rats with radiation proctitis. [Cytotherapy] Abstract

Mesenchymal Stromal Cells Are Genetically Stable under a Hostile In Vivo-Like Scenario as Revealed by In Vitro Micronucleus Test
Scientists exposed umbilical cord mesenchymal stromal cells (MSCs) and placenta-derived MSCs to diverse conditions simulating an in vivo scenario and studied their genetic stability by use of an in vitro micronucleus test. [Cytotherapy] Abstract

Peripheral Blood Mononuclear Cells Enhance Cartilage Repair in In Vivo Osteochondral Defect Model
Investigators characterized peripheral blood mononuclear cells in terms of their potential in cartilage repair and investigated their ability to improve the healing in a pre-clinical large animal model. [PLoS One] Full Article

Development of Novel AAV Serotype 6 Based Vectors with Selective Tropism for Human Cancer Cells
Scientists developed chimeric AAV vectors with Arg-Gly-Asp (RGD) peptide incorporated into the viral capsid to enable specific targeting of integrin-overexpressing malignant cells. These AAV6-RGD vectors improved transduction efficiency approximately three-fold compared with wild-type AAV6 vectors by enhancing the viral entry into the cells. [Gene Ther] Abstract

High Rate of Hematological Responses to Sorafenib in FLT3-ITD Acute Myeloid Leukemia Relapsed after Allogeneic Hematopoietic Stem Cell Transplantation
Researchers retrospectively collected data on the off-label use of the tyrosine-kinase inhibitor sorafenib, either alone or in association with hypomethylating agents and adoptive immunotherapy, in 13 patients with post-transplantation Flt3-ITD positive acute myeloid leukemia relapses. [Eur J Haematol] Abstract

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Controlled Release Strategies for Modulating Immune Responses to Promote Tissue Regeneration
Focusing on targeted immuno-modulation, the authors describe local delivery techniques to sites of inflammation as well as systemic approaches that preferentially target subsets of immune populations. [J Control Release] Abstract

Coming to TERMs with Tissue Engineering and Regenerative Medicine in the Lung
Investigators capture and review the current state of the art in lung bioengineering, from the multimodal approaches to creating anatomically appropriate lung scaffolds that can be recellularized to eventually yield functioning, transplant-ready lungs. [Am J Physiol Lung Cell Mol Physiol] Abstract

Preventing Stem Cell Transplantation-Associated Viral Infections using T-Cell Therapy
The authors focus on the immunotherapeutic strategies that have been used to prevent and treat infections over the past 20 years and outlines different refinements that have been introduced with the goal of moving this therapy beyond specialized academic centers. [Immunotherapy] Abstract

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

Free Wallchart from Nature Reviews Neuroscience on Modeling Neurological Disease with iPS Cells
Pivotal Phase II Study Showed Roche’s Investigational Immunotherapy Atezolizumab Shrank Tumors in People with a Specific Type of Lung Cancer
Roche announced that in the large pivotal Phase II study, BIRCH, the investigational cancer immunotherapy atezolizumab met its primary endpoint and shrank tumors in people with locally advanced or metastatic non-small cell lung cancer whose disease expressed programmed death ligand-1. [F. Hoffmann-La Roche Ltd.] Press Release

Kite Pharma Provides Update on KTE-C19 Clinical Trial
Kite Pharma, Inc. provided an update from the company’s ongoing Phase I/II clinical trial of KTE-C19 in patients with refractory aggressive non-Hodgkin’s lymphoma who have failed prior chemotherapy treatments and have a poor prognosis. There was one patient death early in the study, which was determined to be unrelated to KTE-C19 by the study investigator. [Press release from Kite Pharma, Inc. discussing research to be presented at the 2015 American Society of Hematology (ASH) Annual Meeting, Orlando] Press Release

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We Need a National Debate on Science
Canada’s commitment to science, and our scientific capacity, made us an international leader for years. It was Canadian medical researchers who decoded the breast cancer genome, invented medical insulin and have developed a promising Ebola vaccine. Given the clear importance of science in our lives, why has a question about science policy never — not once — been asked in a federal leaders’ debate? [Toronto Star] Editorial

Cuts to MD/PhD Funding Greeted with ‘Horror’ by Medical Scientists
Canada’s medical research community is reacting with shock and disappointment to the cancellation of a 30-year program to train doctors who see patients and work as scientists searching for new treatments. [Canadian Broadcasting Corporation] Editorial

Apparent ‘Pay to Cite’ Offer Sparks Internet Outrage
Scientists are erupting in outrage on this otherwise sleepy August day. The cause? Cyagen, a purveyor of transgenic research mice, is seemingly offering its scientist customers cash if they cite one of their products in a published paper. [ScienceInsider] Editorial

Animal Advocacy Group Targets Cat and Dog Research Using Novel Crowdsourcing Campaign
Using an unusual crowdsourcing technique to generate hundreds of public records requests, an animal advocacy group claims it has uncovered evidence that an Ohio State University lab has violated National Institutes of Health rules concerning the use of dogs in biomedical research. [ScienceInsider] Editorial

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Merck and MD Anderson Announce Immuno-Oncology Research Collaboration in Solid Tumors
Merck and The University of Texas MD Anderson Cancer Center announced a strategic clinical research collaboration to evaluate Merck’s anti-PD-1 therapy, KEYTRUDA®, in combination with other treatments, such as chemotherapy, radiation therapy and/or novel anti-tumor medicines. [The University of Texas MD Anderson Cancer Center] Press Release

Sorrento Therapeutics and Its Wholly-Owned Subsidiary, TNK Therapeutics, to Exclusively License CytoLumina’s NanoVelcro Circulating Tumor Cell Profiling Assay
Sorrento Therapeutics, Inc. announced that it as well as its wholly-owned subsidiary TNK Therapeutics, Inc. have entered into a binding term sheet to exclusively license the NanoVelcro circulating tumor cell profiling assay technology from CytoLumina Technologies Corp. and FetoLumina Technologies Corp. [Sorrento Therapeutics, Inc.] Press Release

Mallinckrodt Pharmaceuticals to Expand Hospital Growth Platform with Acquisition of Therakos, Inc.
Mallinckrodt plc and the Gores Group announced that they have entered into a definitive agreement under which a subsidiary of Mallinckrodt will acquire Therakos Inc., a leading immunotherapy company in a transaction valued at approximately $1.325 billion. [Mallinckrodt plc] Press Release

UCI-Led Team Begins First Clinical Trial of Stem Cell-Based Retinitis Pigmentosa Treatment
Participants are being enrolled in the first clinical trial that tests the use of retinal progenitor cells to treat retinitis pigmentosa, reported project director Dr. Henry Klassen of UCI’s Gavin Herbert Eye Institute and Sue & Bill Gross Stem Cell Research Center. [University of California, Irvine] Press Release

Regenerating Nerve Tissue in Spinal Cord Injuries
Researchers at Rush University Medical Center are exploring a new therapy using stem cells to treat spinal cord injuries within the first 14 to 30 days of injury. [Rush University Medical Center] Press Release

Japan Grants Safety Clearance to Pluristem’s PLX-PAD Cells for Use in Clinical Trials
Pluristem Therapeutics Inc. announced that Japan’s Pharmaceuticals and Medical Devices Agency has cleared the company’s PLX-PAD cells for use in clinical trials in Japan. [Pluristem Therapeutics Inc.] Press Release

Avalanche Biotechnologies, Inc. Reports Second Quarter 2015 Financial Results and Provides Update on AVA-101 Program
Avalanche Biotechnologies, Inc. reported that after further analyses of results from a previously reported Phase IIa trial of AVA-101 for the potential treatment of wet age-related macular degeneration, it will not initiate a Phase IIb clinical trial in the second half of 2015. [Avalanche Biotechnologies, Inc.] Press Release

Cancer Research Institute to Honor Outstanding Contributions to Basic and Tumor Immunology Research
The Cancer Research Institute will bestow its highest honors on three scientists who have made fundamental contributions to the fields of immunology and cancer immunotherapy. [Cancer Research Institute] Press Release
National Institute on Deafness and Other Communication Disorders; Notice of Closed Meetings (FR Doc. No: 2015-20097)

Government-Owned Inventions; Availability for Licensing (FR Doc. No: 2015-19912)
Rare Diseases: Common Issues in Drug Development Guidance for Industry
Food and Drug Administration (United States)

Rare Diseases: Common Issues in Drug Development; Draft Guidance for Industry; Availability (FR Doc. No: 2015-20235)

Guidance for Entities Considering Whether To Register as Outsourcing Facilities Under Section 503B of the Federal Food, Drug, and Cosmetic Act; Guidance for Industry; Availability (FR Doc. No: 2015-19740)
NEW The 15th International Conference on Progress in Vaccination Against Cancer (PIVAC-15)
October 6-8, 2015
Tübingen, Germany

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Senior Research Technician (Qu Biologics Inc.)

Postdoctoral Fellow Position – Genome Editing, Synthetic Biology, and Cell Evolution and Engineering (University of California – Irvine)

Research Associate – Cell Biology (Editas Medicine)

Senior Research Associate (Editas Medicine)

Postdoctoral Fellow – Biotechnology (Qu Biologics Inc.)

Cell Biology Lab Manager (Harvard Apparatus Regenerative Technology)

Science Writer – Cardiovascular Regeneration (Houston Methodist Research Institute)

PhD Studentship – Veterinary and Animal Sciences (University of Milan)

Postdoctoral Research Associate – Breast Stem Cell Biology (University of Miami Miller School of Medicine)

Aseptic Manufacturing Associate (Lonza Biologics, Inc.)

Postdoctoral Fellow – Stem Cell Biology (INSERM)

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