Volume 17.12 | Mar 28

Issue 17.12 March 28, 2016
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TOP STORY
Stem Cells Used to Successfully Regenerate Damage in Corticospinal Injury
Researchers report that they have successfully directed stem cell-derived neurons to regenerate lost tissue in damaged corticospinal tracts of rats, resulting in functional benefit. Specifically, the researchers grafted multipotent neural progenitor cells into sites of spinal cord injury in rats. [Press release from the University of California, San Diego School of Medicine discussing online prepublication in Nature Medicine] Press Release | Abstract
Register Now: Webinar on Using iPSCs for Studying Pancreatic Development and Disease
 
PUBLICATIONS (Ranked by impact factor of the journal)
Targeted Fibrillar Nanocarbon RNAi Treatment of Acute Kidney Injury
Scientists described an ammonium-functionalized carbon nanotube–mediated transport of small interfering RNA selectively and with high efficiency to renal proximal tubule cells in animal models of acute kidney injury. [Sci Transl Med] Abstract

Enhanced Cancer Immunotherapy by Microneedle Patch-Assisted Delivery of Anti-PD1 Antibody
Investigators report an innovative self-degradable microneedle patch for the sustained delivery of anti-PD-1 in a physiologically controllable manner. [Nano Lett] Abstract | Press Release

Inhibition of IL-1R1/MyD88 Signaling Promotes Mesenchymal Stem Cell-Driven Tissue Regeneration
Because Toll-like receptor and interleukin-1 receptor, type 1 (IL-1R1) ligands have been shown to influence the repair process of various tissues, scientists explored their role during bone regeneration, seeking to design regenerative strategies integrating a control of their signaling. [Nat Commun] Full Article

Beta-Tricalcium Phosphate Granules Improve Osteogenesis In Vitro and Establish Innovative Osteo-Regenerators for Bone Tissue Engineering In Vivo
Investigators aimed to explore suitable β-tricalcium phosphate granules for bone regeneration and identified an efficient method to establish β-TCP-based osteo-regenerators. [Sci Rep] Full Article

A Small Indel Mutant Mouse Model of Epidermolytic Palmoplantar Keratoderma and Its Application to Mutant-Specific shRNA Therapy
Researchers report a knock-in transgenic mouse model that carried a small insertion–deletion mutant of Krt9, c.434delAinsGGCT, corresponding to the human mutation KRT9/c.500delAinsGGCT, which resulted in a human epidermolytic palmoplantar keratoderma (EPPK)-like phenotype in the weight-stress areas of the fore- and hind-paws of both Krt9+/mut and Krt9mut/mut mice. Then, they developed a mutant-specific short hairpin RNA (shRNA) therapy for EPPK mice. [Mol Ther Nucleic Acids] Full Article

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington’s Disease
Data demonstrated strong allele-selective silencing of mutated huntingtin (HTT) by miSNP50 targeting rs362331 and total HTT silencing by miH12 both in vitro and in vivo. [Mol Ther Nucleic Acids] Full Article

Muscle Tissue Engineering Using Gingival Mesenchymal Stem Cells Encapsulated in Alginate Hydrogels Containing Multiple Growth Factors
The authors developed an injectable 3D RGD-coupled alginate scaffold with multiple growth factor delivery capacity for encapsulating gingival mesenchymal stem cells (GMSCs), and to evaluate the capacity of encapsulated GMSCs to differentiate into myogenic tissue in vitro and in vivo where encapsulated GMSCs were transplanted subcutaneously into immunocompromised mice. [Ann Biomed Eng] Full Article

Using Primate Neural Stem Cells Cultured in Self-Assembling Peptide Nanofiber Scaffolds to Repair Injured Spinal Cords in Rats
Primate neural stem cells were cultured in self-assembling peptide nanofiber scaffolds to repair spinal cord injury. [Spinal Cord] Abstract

Expression of DAI by an Oncolytic Vaccinia Virus Boosts the Immunogenicity of the Virus and Enhances Antitumor Immunity
Investigators developed an oncolytic vaccinia virus that expresses intracellular pattern recognition receptor DNA-dependent activator of IFN-regulatory factors to boost the innate immune system and to activate adaptive immune cells in the tumor. [Mol Ther Oncolytics] Full Article

Weekend-Free Culture hPSCs with mTeSR™1 or TeSR™-E8™
 
REVIEWS
Driving CAR T-Cells Forward
The authors discuss trials and present strategies that can increase the antitumor efficacy and safety of CAR T-cell therapy. Given the fast-moving nature of this field, they only discuss studies with direct translational application currently or soon-to-be tested in the clinical setting. [Nat Rev Clin Oncol] Abstract

Induced Pluripotent Stem Cells: At the Heart of Cardiovascular Precision Medicine
The authors summarize the progress that has been made in the field of human induced pluripotent stem cell (hiPSC) technology, with particular emphasis on cardiovascular disease modelling and drug development. The growing roles of hiPSC technology in the practice of precision medicine are also discussed. [Nat Rev Cardiol] Abstract

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

Learn more about STEMvision automated mouse hematopoietic colony-forming unit (CFU) assay imaging and counting.
 
SCIENCE NEWS
Fujifilm Achieves Major Improvements in Bone Regeneration Capability through Transplants of the Matrix to Areas of Bone Defect
Fujifilm Corporation has succeeded in significantly improving bone regeneration capability through the transplantation, to areas of defect in the calvarial bone of rats, of cellnest™ recombinant peptide based on human collagen type I, an extracellular matrix required for the cell cultures used in regenerative medicine. [Press release from Fujifilm Corporation discussing research presented at the 15th Congress of the Japanese Society for Regenerative Medicine, Osaka] Press Release

RXi Pharmaceuticals to Present at the Oligonucleotide Therapeutics and Delivery Conference
RXi Pharmaceuticals Corporation announced that Dr. Pamela Pavco, Chief Development Officer at RXi Pharmaceuticals, will present an overview of the company’s dermal and ocular clinical programs with RXI-109. [Press release from RXi Pharmaceuticals Corporation discussing research to be presented at the Oligonucleotide Therapeutics and Delivery Conference, Cambridge] Press Release

Kolltan Pharmaceuticals to Present Preclinical Data
Kolltan Pharmaceuticals, Inc. announced oral and poster presentations of key preclinical data relating to certain of its drug development programs, including KTN3379, KTN0158, ALK-ADC and MET. [Press release from Kolltan Pharmaceuticals, Inc. (Business Wire) discussing research to be presented at American Association for Cancer Research (AACR), New Orleans] Press Release

Join us for Advances in Immuno-oncology 2016 in London!
 
POLICY
Canadian Scientists Smile as Liberals Deliver a Déjà Vu Budget
Canada’s research community finds itself stepping into the sunshine after the nation’s new Liberal government unveiled a fiscal blueprint for 2015–16 that provides an immediate $72.79 million per year injection into the budgets of the nation’s research granting councils. The new money comes after years of static or declining budgets. [ScienceInsider] Editorial

House Budget Plan Would Rearrange and Restrict Federal Research Portfolio
If an influential congressional budget committee has its way, the U.S. Department of Commerce could be eliminated, and many research programs at the National Science Foundation and the Department of Energy would be sharply curtailed. Those startling changes in federal research policy are part of a blueprint for spending that was released yesterday by the budget committee of the U.S. House of Representatives. [ScienceInsider] Editorial

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BUSINESS
Quest PharmaTech’s Subsidiary, OncoQuest, Signs Agreement with Shenzhen Hepalink Pharmaceutical Co., Ltd. to Fund Immunotherapy for Treatment of Cancer in China
Quest PharmaTech Inc. announces that its subsidiary, OncoQuest Inc. has signed an agreement with Shenzhen Hepalink Pharmaceutical Co., Ltd., to fund the research and development of immunotherapies for treatment of cancer in China. [Quest PharmaTech Inc.] Press Release

ORIG3N and Sharp Edge Labs Enter into Drug Discovery Alliance
ORIG3N, Inc. and Sharp Edge Labs, Inc. announced that they have entered into a drug discovery alliance. As part of the alliance, ORIG3N will make patient-derived cells available to Sharp Edge Labs for use in their drug discovery platform for genetic disorders. [ORIG3N, Inc. (PR Newswire Association LLC)] Press Release

Stem Cell Researcher Tracy Grikscheit Awarded $7.1 Million by CIRM
Tracy C. Grikscheit, a principal investigator with USC Stem Cell and The Saban Research Institute of Children’s Hospital Los Angeles, has received a $7.1 million grant from the California Institute of Regenerative Medicine Translational Research (CIRM) program to develop a cellular therapy for the treatment of nerve disorders of the digestive system. [Keck School of Medicine] Press Release

CIRM Grant to Fund Proposed Stem Cell Trials for ALS
The Independent Citizens Oversight Committee of the California Institute for Regenerative Medicine (CIRM) approved a $6.3 million grant to a research team from the University of California, San Diego School of Medicine and University of California, Davis to pursue a novel human embryonic stem cell-based therapy to rescue and restore neurons devastated by amyotrophic lateral sclerosis or ALS. [University of California, San Diego] Press Release

Three ERC-Grants for Research Groups at the Vienna Biocenter
Jan-Michael Peters and Tim Clausen of the Research Institute of Molecular Pathology and Jürgen Knoblich of the Institute of Molecular Biotechnology each receive an Advanced Grant by the European Research Council ERC. [Institute of Molecular Pathology] Press Release

Ultragenyx Funds Researcher’s Quest to Treat His Daughter’s Disease
SLU’s Center for World Health and Medicine, has entered into a three-year collaboration to advance a potential treatment identified by Fran Sverdrup for facioscapulohumeral muscular dystrophy, a disease that causes muscle degeneration. [Saint Louis University] Press Release

RetroSense Therapeutics Doses First Patient in Phase I/II Clinical Trial for Lead Compound RST-001
RetroSense Therapeutics LLC announced that the first patient has been successfully dosed in the first clinical trial to evaluate the safety of RST-001. RST-001 is designed to restore some vision in retinitis pigmentosa, a genetic condition that leads to the progressive degeneration of rod and cone photoreceptors, resulting in severe vision loss and blindness. [RetroSense Therapeutics LLC (Business Wire)] Press Release

ACell®, Inc. Receives FDA Clearance for Concurrent Use of Its Wound Management Devices
ACell, Inc. announced that it has received additional FDA clearance for its MicroMatrix® device, which describes the concurrent use of MicroMatrix® in conjunction with Cytal™ Wound Matrix or Cytal Burn Matrix devices. [ACell, Inc.] Press Release

Regen BioPharma, Inc. Submits IND Application to FDA on tCellVax, a Checkpoint Inhibitor for Cancer Therapy
Regen BioPharma, Inc. announced submission to the U.S. Food and Drug Administration (FDA) of an Investigational New Drug application focused on using a novel method to unleash the body’s innate ability to kill cancer. [Regen BioPharma, Inc. (PR Newswire Association LLC)] Press Release

UC San Diego Health Joins National Clinical Trial on Hemophilia B Gene Therapy
Thee Hemophilia and Thrombosis Treatment Center at University of California (UC) San Diego Health has joined a nationwide clinical trial testing a potential gene therapy that may one day provide a better and long-lasting treatment for people with hemophilia B. [University of California San Diego] Press Release

Southern Research Scientists Develop an Assay to Find a Cure for Zika
Southern Research announced the development of a unique antiviral assay – a test that can be used by researchers worldwide to detect the virus in infected cell cultures. In addition, Southern Research is working to develop in vivo models that can be used to evaluate vaccines and therapeutic approaches aimed at preventing the further spread of Zika virus disease. [Southern Research] Press Release

IMP-Researcher Johannes Zuber Wins German Cancer Prize
Johannes Zuber, group leader at the research Institute of Molecular Pathology (IMP), receives the German Cancer Prize 2016 in the category of experimental cancer research. The German Cancer Prize is one of the most prestigious scientific awards in the German speaking world and annually honors researchers for groundbreaking discoveries in the field of oncology. [Institute of Molecular Pathology] Press Release
 
NIH
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2016-06868)
 
CBER
March 16, 2016 Approval Letter – KOVALTRY
 
REGULATORY
Food and Drug Administration (United States)

Investigational New Drug Applications for Biological Products; Bioequivalence Regulations; Technical Amendment (FR Doc. No: 2016-06886)

Authorization of Emergency Use of an In Vitro Diagnostic Device for Diagnosis of Zika Virus Infection; Availability (FR Doc. No: 2016-06888)
 
EVENTS
NEW World Cord Blood Congress Europe 2016
May 18-19, 2016
London, United Kingdom

NEW The Stem Cell Niche 2016
May 22-26, 2016
Hillerod, Denmark

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
 
JOB OPPORTUNITIES
NEW Postdoctoral Fellow – Non-Coding RNAs in Malignant Progression (The University of Texas MD Anderson Cancer Center)

NEW Postdoctoral Associate – Stem Cell Biology (University of Illinois Chicago College of Medicine)

NEW Postdoctoral Associate – Genetic Medicine (Cornell University)

NEW Postdoctoral Fellow – Retinal Disease Genomics and Gene Therapy (Ocular Genomics Institute, Mass Eye and Ear and Harvard Medical School)

Scientist – Human Pluripotent Stem Cell Neural Differentiation (STEMCELL Technologies Inc.)

Postdoctoral Fellow – T Cell Reconstitution (Fred Hutchinson Cancer Research Center)

Postdoctoral Fellow – Self Organization of the Pluripotent Lineage (Max-Planck-Institute for Molecular Biomedicine)

Associate Scientist – Hematopoietic Stem Cell Biology (Editas Medicine)

Research Associate – Hematopoietic Stem Cell Biology (Editas Medicine)

Postdoctoral Fellowship – Adeno-Associated Virus Gene Therapy (Icahn School of Medicine at Mount Sinai)

Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital)

Postdoctoral Fellowship – Stem Cells, Oligodendrogenesis and Remyelination (UC Davis)

PhD Positions – Cancer, Microbiology and Stem Cell Engineering (Virginia Commonwealth University)

PhD Positions – Tendon Repair and Regeneration (NUI Galway)

PhD Position – Leukemogenesis and Treatment Resistance (Institute for Tumor Biology and Experimental Therapy)

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

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

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


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