Volume 17.05 | Feb 8

Issue 17.05 February 8, 2016
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Curing Disease by Repairing Faulty Genes
Scientists developed a way to deliver the CRISPR genome repair components more efficiently than previously possible, and they also believe it may be safer for human use. [Press release from Massachusetts Institute of Technology discussing online prepublication in Nature Biotechnology] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Cre-Dependent Selection Yields AAV Variants for Widespread Gene Transfer to the Adult Brain
Investigators describe a capsid selection method, called Cre recombination–based adeno-associated virus (AAV) targeted evolution (CREATE), that enables the development of AAV capsids that more efficiently transduce defined Cre-expressing cell populations in vivo. [Nat Biotechnol] Abstract | Press Release

Implantation of Healthy Matrix-Embedded Endothelial Cells Rescues Dysfunctional Endothelium and Ischemic Tissue in Liver Engraftment
Investigators sought to understand how endothelial state determines liver recovery after hepatectomy and engraftment. Healthy matrix-embedded endothelial cells created a functional vascular splice in donor and recipient liver after 70% hepatectomy in mouse protecting these livers from ischemic injury, hepatic congestion and inflammation. [Gut] Abstract

Therapeutically Engineered Induced Neural Stem Cells Are Tumor-Homing and Inhibit Progression of Glioblastoma
Scientists provide evidence that transdifferentiation-derived induced neural stem cells (iNSCs) are an efficacious therapeutic strategy for brain cancer. They found that iNSCs genetically engineered with optical reporters and tumoricidal gene products retain the capacity to differentiate and induced apoptosis in co-cultured human glioblastoma cells. [Nat Commun] Full Article

Transplanted Neurons Integrate into Adult Retinas and Respond to Light
Researchers investigated whether transplanted retinal ganglion cells (RGCs) can integrate into the mature retina. Transplanted RGCs acquired the general morphology of endogenous RGCs, with axons orienting towards the optic nerve head of the host retina and dendrites growing into the inner plexiform layer. [Nat Commun] Full Article

Enhanced Renoprotective Effect of IGF-1 Modified Human Umbilical Cord-Derived Mesenchymal Stem Cells on Gentamicin-Induced Acute Kidney Injury
Scientists investigated whether insulin-like growth factor-1 (IGF-1)-modified umbilical cord-derived mesenchymal stem cells hold an enhanced protective effect on gentamicin-induced acute kidney injury in vivo. [Sci Rep] Full Article

In Vivo Transplantation of Enteric Neural Crest Cells into Mouse Gut; Engraftment, Functional Integration and Long-Term Safety
Researchers investigated the potential of enteric neural stem cell therapy approaches for gastrointestinal disorders by transplanting mouse enteric neural crest cells into ganglionic and aganglionic mouse gut in vivo and analyzing functional integration and long-term safety. [PLoS One] Full Article

Efficacy and Safety of Myocardial Gene Transfer of Adenovirus, Adeno-Associated Virus and Lentivirus Vectors in the Mouse Heart
To find the most suitable and safe vector for cardiac gene transfer, researchers delivered adenovirus, adeno-associated virus and lentivirus vectors into the mouse heart with sophisticated closed-chest echocardiography-guided intramyocardial injection method for comparing them with regards to transduction efficiency, myocardial damage, effects on the left ventricular function and electrocardiography. [Gene Ther] Abstract

Downregulation of Thymidylate Synthase by RNAi Molecules Enhances the Antitumor Effect of Pemetrexed in an Orthotopic Malignant Mesothelioma Xenograft Mouse Model
Researchers investigated whether non-viral anti-thymidylate synthase (TS) RNA interference (RNAi) embedded liposome would effectively guide the downregulation of TS in human malignant mesothelioma MSTO-211H cells. They found it enhanced the antitumor effect of pemetrexed both in vitro and in vivo. [Int J Oncol] Abstract

Treatment of Hypophosphatasia by Muscle-Directed Expression of Bone-Targeted Alkaline Phosphatase via Self-Complementary AAV8 Vector
Investigators constructed a self-complementary type 8 adeno-associated virus (AAV) vector that expresses tissue-nonspecific alkaline phosphatase with deca-aspartates at the C-terminus via the muscle creatine kinase promoter and examined the efficacy of muscle-directed gene therapy. [Mol Ther Methods Clin Dev] Full Article

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Stem and Progenitor Cell-Based Therapy of the Central Nervous System: Hopes, Hype, and Wishful Thinking
The author seeks to define neurological conditions most appropriate for cell replacement therapy by considering its potential efficacy and clinical feasibility in those disorders, as well as potential impediments to its application. [Cell Stem Cell] Abstract

Mesenchymal Stromal Cells in Renal Transplantation: Opportunities and Challenges
The findings of preclinical and initial clinical studies support the potential tolerance-inducing effects of mesenchymal stromal cells (MSCs) and highlight the unanticipated complexity of MSC therapy in kidney transplantation. [Nat Rev Nephrol] Abstract

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

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Immune Therapy for Brain Tumors: A New Promising Avenue
A new two-drug combination, currently in early clinical trials, might help people diagnosed with glioblastoma to fight the disease. Scientists reported that a large number of study participants responded well to the drug combination. [Press release from the University of New Mexico discussing research presented at the 20th Annual Scientific Meeting of the Society for Neuro-Oncology (SNO), San Antonio] Press Release

Study on Use of Umbilical Cord vs. Biocellulose Film for Antenatal Spina Bifida Repair, Regenerative Patch May Improve Neurological Outcomes
Researchers presented findings from a study titled, “Cryopreserved Human Umbilical Cord vs. Biocellulose Film for Antenatal Spina Bifida Repair.” [Press release from the Society for Maternal-Fetal Medicine (SMFM) discussing research presented at the The Pregnancy Meeting™, Atlanta] Press Release

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White House Proposes $1 Billion for Cancer ‘Moonshot’
The White House will propose spending close to $1 billion over the next two years on a “moonshot” to fight cancer. President Barack Obama will ask Congress for $755 million for fiscal year 2017, and will try to spend $195 million this year to get things started. [NBC News] Editorial

Worries about Brain Damage in Infants Linked to Zika Leads WHO to Declare a Public Health Emergency
Increasingly strong links between the spread of Zika virus and microcephaly in newborns led the World Health Organization (WHO) to declare a Public Health Emergency of International Concern. [ScienceInsider] Editorial

Germany’s Excellence Program Gets Good Grades
Germany should award millions of euros in extra funding to its ten top-performing universities, an international commission recommended. That is one of the best ways to build on the country’s Excellence Initiative, a decade-long program that was supposed to boost German universities to world-class status. [ScienceInsider] Editorial

Biotech Giant Publishes Failures to Confirm High-Profile Science
A biotechnology firm is releasing data on three failed efforts to confirm findings in high-profile scientific journals — details that the industry usually keeps secret. [Nature News] Editorial

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Adaptimmune and GSK Expand Strategic Immunotherapy Collaboration
Adaptimmune Therapeutics plc and GlaxoSmithKline plc (GSK) announced that the companies have expanded the terms of their strategic collaboration agreement to accelerate Adaptimmune’s lead clinical cancer program, an affinity enhanced T-cell immunotherapy (GSK3377794) targeting NY-ESO-1, toward pivotal trials in synovial sarcoma. [Adaptimmune Therapeutics plc] Press Release

Dana-Farber Cancer Institute’s Belfer Center Announces Immuno-Oncology Research Collaboration with Array BioPharma
Dana-Farber Cancer Institute announced an immuno-oncology collaboration with potential applicability in a wide range of oncology indications with Array BioPharma. The research team at Dana-Farber will work with Array scientists on novel immune-oncology targets with the goal of bringing innovative medicines to patients. [Dana-Farber Cancer Institute] Press Release

Dream Team Gets $11.7 Million to Fight Brain Cancers
A “Dream Team” of top Canadian scientists, led by two outstanding figures in stem cell research, is launching a new attack on brain cancers. The team will receive $11.7 million over four years from Stand Up To Cancer Canada, Genome Canada, the Canadian Institutes of Health Research, the Cancer Stem Cell Consortium, and Ontario Institute for Cancer Research. [Canadian Stem Cell Foundation] Press Release

NUI Galway Launch €6 Million Robotic Stem Cell Production Project
NUI Galway’s Regenerative Medicine Institute has launched a new €6 million research project AUTOSTEM to develop pioneering manufacturing systems for stem cell therapy. Funded by the EU Horizon 2020 program, AUTOSTEM will go a long way towards meeting the demand for these new treatments, by transforming the way stem cells are manufactured. [National University of Ireland, Galway] Press Release

With $3 Million Award, HSU Generates Stem Cell Scholars
Humboldt State University (HSU) biology professors Jacob Varkey and Amy Sprowles are among educators at 12 universities to secure funding from the California Institute for Regenerative Medicine’s Bridges 2.0 Training Program. The award of $3,044,950 will fund stem cell training for HSU students and support up to ten paid research internships annually for the next five years. [Humboldt State University] Press Release

NIH Awards $2.8 Million to FAU Researchers and Collaborators
The National Eye Institute of the National Institutes of Health (NIH) has awarded a $2.8 million, multi-institution grant to define the mechanisms that govern how cells decide whether to become a mature cell or whether to die. [Florida Atlantic University] Press Release

Terry Fox Award Helps Ottawa Researcher Develop Next-Generation Cancer Immunovirotherapy
The Terry Fox Research Institute has awarded Dr. Guy Ungerechts (The Ottawa Hospital and the University of Ottawa) a $450,000 New Investigator grant to develop innovative new treatments to destroy cancer cells and stimulate a patient’s own immune system to attack tumors, while leaving healthy tissues unharmed. [Terry Fox Research Institute] Press Release

Regenerative Medicine Awards Lay Groundwork for Therapies of Tomorrow
Two collaborative teams within USC Stem Cell are developing new approaches that could eventually help patients with heart disease, frontotemporal dementia and amyotrophic lateral sclerosis. Totaling $140,000 each, the awards support USC-affiliated faculty members pursuing multi-investigator research collaborations with the goal of curing diseases using stem cells as tools. [University of Southern California] Press Release

New Department of Defense Grant Funds Efforts to Treat ALS with Stem Cells
Evan Snyder, M.D., Ph.D. was awarded a grant to develop a stem cell treatment for amyotrophic lateral sclerosis (ALS). This grant will support research on human neural stem cells as an approach to support the survival and function of existing motor neurons. [Sanford Burnham Prebys Medical Discovery Institute] Press Release

OSE Pharma Announces U.S. Initiation of Atalante 1, the Company’s Global, Pivotal Phase III Trial for Tedopi® Immunotherapy in Non-Small Cell Lung Cancer
OSE Pharma SA announced initiation in the United States of its Phase III clinical trial, Atalante 1. This global, pivotal trial will evaluate Tedopi®, the company’s lead product, for advanced non-small cell lung cancer. [OSE Pharma SA] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2016-02188)

National Institute of Neurological Disorders and Stroke; Notice of Closed Meeting (FR Doc. No: 2016-02182)
Enforcement Policy on National Health Related Item Code and National Drug Code Numbers Assigned to Devices; Draft Guidance for Industry and Food and Drug Administration Staff; Availability and Request for Comments
Food and Drug Administration (United States)

Human Factors Studies and Related Clinical Study Considerations in Combination Product Design and Development; Draft Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc. No: 2016-01888)
NEW Malaysian Association for Cell Therapy (MACT) 2016 Scientific Symposium
April 2, 2016
Kuala Lumpur, Malaysia

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Positions – Gene Regulation and Translational Cancer Research (University of Texas Health Science Center)

Postdoctoral Position – Hematology/Oncology (Penn State College of Medicine)

Group Leader – Cell Therapy (Miltenyi Biotec)

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)

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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