Volume 17.08 | Feb 29

Issue 17.08 February 29, 2016
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Telomerase Gene Therapy Rescues Telomere Length, Bone Marrow Aplasia and Survival in Mice with Aplastic Anemia
Researchers tested the therapeutic efficacy of telomerase activation by using AAV9 gene therapy vectors carrying the telomerase Tert gene in two independent mouse models of aplastic anemia owing to short telomeres. [Blood] Abstract | Press Release
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PUBLICATIONS (Ranked by impact factor of the journal)
BPOZ-2 Gene Delivery Ameliorates Alpha-Synucleinopathy in A53T Transgenic Mouse Model of Parkinson’s Disease
Researchers report that lentiviral administration of bpoz-2 gene indeed lowers the burden of alpha-synuclein in DA neurons in the nigra of A53T transgenic mouse. [Sci Rep] Full Article

Repair of Liver Mediated by Adult Mouse Liver Neuro-Glia Antigen 2-Positive Progenitor Cell Transplantation in a Mouse Model of Cirrhosis
NG2-expressing cells are a population of periportal vascular stem/progenitors that were isolated from healthy adult mouse liver by using a “Percoll-Plate-Wait” procedure. The authors demonstrated that isolated cells are able to restore liver function after transplantation into a cirrhotic liver, and co-localized with the pericyte marker and CK19. [Sci Rep] Full Article

Cardiomyocytes Derived from MHC-Homozygous Induced Pluripotent Stem Cells Exhibit Reduced Allogeneic Immunogenicity in MHC-Matched Non-Human Primates
Scientists transplanted fluorescence-labeled induced pluripotent stem cell-cardiomyocytes donated from a macaque with homozygous major histocompatibility complex (MHC) haplotypes into the subcutaneous tissue and hearts of macaques having heterozygous MHC haplotypes or without identical MHC alleles in conjunction with immune suppression. [Stem Cell Reports] Full Article | Graphical Abstract

An Endochondral Ossification-Based Approach to Bone Repair: Chondrogenically-Primed MSC-Laden Scaffolds Support Greater Repair of Critical-Sized Cranial Defects than Osteogenically Stimulated Constructs In Vivo
Scientists hypothesised that stimulation of mesenchymal stem cell (MSC) chondrogenesis and subsequent hypertrophy within collagen-based scaffolds would lead to improved vascularisation and bone formation when implanted within a critical-sized bone defect in vivo. [Tissue Eng Part A] Abstract

Poxvirus-Based Active Immunotherapy with PD-1 and LAG-3 Dual Immune Checkpoint Inhibition Overcomes Compensatory Immune Regulation, Yielding Complete Tumor Regression in Mice
In therapeutic CT26-HER-2 tumor models, MVA-BN-HER2 poxvirus immunotherapy resulted in significant tumor growth delay accompanied by a robust, tumor-infiltrating T cell response that was characterized by low to mid-levels of PD-1 expression on T cells. [PLoS One] Full Article

β1-Na+,K+-ATPase Gene Therapy Upregulates Tight Junctions to Rescue Lipopolysaccharide-Induced Acute Lung Injury
Scientists showed that delivery of a plasmid expressing β1-Na+,K+-ATPase alone or in combination with epithelial sodium channel α1 subunit using electroporation not only protected from subsequent LPS-mediated lung injury, but also treated injured lungs. [Gene Ther] Abstract

Specific Immunotherapy Generates CD8+ CD196+ T Cells to Suppress Lung Cancer Growth in Mice
The authors aimed to inhibit lung cancer (LC) growth in a mouse model by using an LC-specific vaccination. A LC mouse model was created by adoptive transplantation with LC cells. [Immunol Res] Abstract

Transplantation of Autologous Synovial Mesenchymal Stem Cells Promotes Meniscus Regeneration in Aged Primates
Researchers investigated whether transplantation of aggregates of autologous synovial mesenchymal stem cells promoted meniscal regeneration in aged primates. [J Orthop Res] Abstract

Effect of Adenovirus-Mediated RNA Interference of IL-1β Expression on Spinal Cord Injury in Rats
Investigators introduced an adenoviral vector expressing interleukin-1β (IL-1β) small-hairpin RNA into injured spinal cords to evaluate the therapeutic potential of IL-1β downregulation in a rat model of spinal cord injury. [Spinal Cord] Abstract

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iPSCs-Based Anti-Aging Therapies: Recent Discoveries and Future Challenges
The main biological hallmarks of the aging process include stem cell exhaustion and cellular senescence. Consequently, research efforts to treat age-related diseases as well as anti-aging therapies in general have recently focused on potential ‘reprogramming’ regenerative therapies. [Ageing Res Rev] Abstract

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

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Potential Treatment for Huntington’s Disease, Found Effective, Safe in Mice, Monkeys
A drug that would be the first to target the cause of Huntington’s disease is effective and safe when tested in mice and monkeys, according to data. [Press release from the University of British Columbia (EurekAlert!) discussing research to be presented at the American Academy of Neurology’s 68th Annual Meeting, Vancouver] Press Release

Stroke Patients Show Higher Rate of Recovery from MultiStem® Cell Therapy Studied at University Hospitals Case Medical Center
Data presented by Athersys indicated that stroke patients treated by an adult stem cell therapy called MultiStem® did significantly better than others who received a placebo one year after treatment. [Press release from University Hospitals Case Medical Center discussing research presented at the 2016 International Stroke Conference, Los Angeles] Press Release | Video

Late-Breaking Research Shows Promise for Patients with Primary Progressive Multiple Sclerosis
In a randomized, double-blind, placebo-controlled Phase III study, ocrelizumab significantly reduced disease activity in patients with primary progressive multiple sclerosis. [Press release from Newswise, Inc. discussing research presented at Americas Committee for Treatment and Research in Multiple Scleroses (ACTRIMS) Forum 2016, New Orleans] Press Release

Provectus Biopharmaceuticals Announces Data on PV-10 and Co-Inhibitory Blockade to Be Presented
Provectus Biopharmaceuticals, Inc. announced that data on intralesional PV-10 and co-inhibitory blockade in a melanoma model will be presented. [Press release from Provectus Biopharmaceuticals, Inc. discussing research to be presented at the American Association for Cancer Research’s (AACR) Annual Meeting 2016, New Orleans] Press Release

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Confusion Reigns as UK Scientists Face Government ‘Gagging’ Clause
UK scientists may be prevented from arguing for changes in national legislation or policy — if research grants are not exempted from a government ban on the use of public funds for political lobbying. But days after scientists raised the alarm about the government’s anti-lobbying move, the situation is mired in confusion. [Nature News] Editorial

India’s Budget Keeps Dream of Genomics Hub Alive
An ambitious plan to turn India into a world-class centre for genomics research and commercialization received a modest boost on 29 February when the government announced its annual budget. [Nature News] Editorial

NIH’s 10% Set-Aside for AIDS Begins to Slip in 2016
After more than 20 years of holding HIV/AIDS research funding at a fixed 10% of its overall budget, the National Institutes of Health will let that level slip this year. Newly public data on grant funding success rates reflect one impetus for abandoning the set-aside: At many institutes, AIDS grants have been much easier to get than non-AIDS funding, suggesting that officials were struggling to find ways to spend the money. [ScienceInsider] Editorial

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Neon Therapeutics Enters Collaborative Research Agreement with the Netherlands Cancer Institute
Neon Therapeutics announced that the company has entered into a collaborative research agreement with the Netherlands Cancer Institute’s (NKI) Antoni van Leeuwenhoek Hospital and the Amsterdam Biotherapeutics Unit. Under the terms of the agreement, Neon Therapeutics and NKI will combine complementary capabilities to develop personalized T cell therapeutics. [Neon Therapeutics] Press Release

Juno Therapeutics, Seattle Cancer Care Alliance and Fred Hutchinson Cancer Research Center Announce New Immuno-Oncology Clinical Trials Unit
Juno Therapeutics, Inc. and Fred Hutchinson Cancer Research Center announced the creation of a new, best-in-class clinical trials unit dedicated to immuno-oncology. [Juno Therapeutics, Inc.] Press Release

OncBioMune Continuing Phase I Trial, While Initiating Phase II Trial of ProscaVax for Prostate Cancer
OncBioMune Pharmaceuticals, Inc. reported positive results from its Phase I clinical trial of ProscaVax, the company’s novel cancer vaccine for prostate cancer. [OncBioMune Pharmaceuticals, Inc.] Press Release

Asterias Biotherapeutics Announces Successful End-of-Phase II Meeting with FDA for AST-VAC1
Asterias Biotherapeutics, Inc. announced the successful completion of an End-of-Phase II meeting with the U.S. Food and Drug Administration (FDA) for AST-VAC1, its investigational therapy targeting acute myeloid leukemia. [Asterias Biotherapeutics, Inc.] Press Release

Capricor Therapeutics Announces the First Patient with Duchenne Muscular Dystrophy-Related Cardiomyopathy Treated with CAP-1002 in the HOPE-Duchenne Phase I/II Clinical Trial
Capricor Therapeutics, Inc. announced continuing enrollment of, and the treatment of the first patient with Duchenne Muscular Dystrophy-related cardiomyopathy with CAP-1002 in the HOPE-Duchenne Phase I/II clinical trial. [Capricor Therapeutics, Inc.] Press Release

National Institutes of Health’s NIAID Initiating Dose Evaluation Studies in Animal Models of Pluristem’s PLX-R18 in the Treatment of Acute Radiation Syndrome
Pluristem Therapeutics Inc. announced that the National Institute of Allergy and Infectious Diseases (NIAID) will initiate studies in large animals to evaluate dosing for Pluristem’s PLX-R18 as a medical counter measure in the treatment of the hematologic components of Acute Radiation Syndrome. [Pluristem Therapeutics Inc.] Press Release

TapImmune Receives Notice of Allowance on U.S. Patent Claims for PolyStart – Next-Generation T-Cell Vaccine
TapImmune, Inc. announced that it has received a Notice of Allowance from the U.S. Patent and Trademark Office for its patent application entitled, “A chimeric nucleic acid molecule with non-AUG initiation sequences.” The patent claims are for the Company’s proprietary PolyStart™ technology which is a platform for the next-generation vaccine technology for the treatment of cancer or infectious diseases. [TapImmune, Inc.] Press Release

CAR Trials Drive Leukemia and Lymphoma Treatment in New Direction
Researchers at UC San Diego Moores Cancer Center have launched three clinical trials to test the safety and efficacy of a novel cellular-immunotherapy that uses modified T cells – one of the immune system’s primary weapons – to treat three different types of blood cancer that often defy existing therapies. [UC San Diego Health] Press Release

La Jolla Institute Receives $5 Million Gift from Pfizer
La Jolla Institute for Allergy and Immunology has received a $5 million gift, which will help establish the Mission Support for Cancer Immunology and Oncology Research. The Institute will use the majority of the funds to establish the Pfizer Endowed Chair in Cancer Immunology and Oncology and support research on the immune system and oncology. [La Jolla Institute for Allergy and Immunology] Press Release

True North Therapeutics Receives Orphan Drug Designation in the European Union for TNT009 for the Treatment of Autoimmune Hemolytic Anemia, including Cold Agglutinin Disease (CAD)
True North Therapeutics announced that the European Medicines Agency granted Orphan Drug Designation for TNT009 for the treatment of autoimmune hemolytic anemia including CAD, in which autoantibodies target and destroy red blood cells, causing anemia, fatigue and potentially fatal thrombosis. [True North Therapeutics, Inc.] Press Release

TxCell Launches New Manufacturing Process Development Laboratories and Technology Transfer Academy
TxCell SA announced the launch of its new process industrialization laboratories and technology transfer academy facilities in Sophia Antipolis, France. [TxCell SA] Press Release
Center For Scientific Review; Notice of Closed Meetings (FR Doc. No: 2016-04234)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2016-04104)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2016-03644)
Public Hearing; Request for Comments – Draft Guidances Relating to the Regulation of Human Cells, Tissues or Cellular or Tissue-Based Products – Postponed
NEW Applications of the Hematopoietic Progenitor Assay Training Course
March 31-April 1, 2016
Vancouver, Canada

NEW Malaysian Association For Cell Therapy 2016 Scientific Symposium
April 2, 2016
Petaling Jaya, Malaysia

NEW ISSCR 2016 Annual Meeting
June 22-25, 2016
San Francisco, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Scientist – Human Pluripotent Stem Cell Neural Differentiation (STEMCELL Technologies Inc.)

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

NEW 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)

Lead Scientist – Translational Therapy (NHS Blood and Transplant)

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

Postdoctoral Positions – Gene Regulation and Translational Cancer Research (University of Texas Health Science Center)

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

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

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