Cell Therapy News Volume 17.39 | Oct 31 2016

TOP STORY

MicroRNA-21 Preserves the Fibrotic Mechanical Memory of Mesenchymal Stem Cells

Investigators showed that priming on physiologically soft silicone substrates suppresses fibrogenesis and desensitizes mesenchymal stem cells (MSCs) against subsequent mechanical activation in vitro and in vivo, and identified the microRNA miR-21 as a long-term memory keeper of the fibrogenic program in MSCs. [Nat Mater] Abstract

(Ranked by impact factor of the journal)

Characterization and Transplantation of Enteric Neural Crest Cells From Human Induced Pluripotent Stem Cells

Human induced pluripotent stem cells (hiPSCs) were induced to differentiate into neural crest stem cells (NCSCs) efficiently. When co-cultured with smooth muscle layers of ganglionic gut tissue, the NCSCs differentiated into different subtypes of mature enteric-like neurons expressing nitric oxide synthase, vasoactive intestinal polypeptide, choline acetyltransferase or calretinin with typical electrophysiological characteristics of functional neurons. [Mol Psychiatry] Full Article

In Vivo Correction of Anemia in β-Thalassemic Mice by γPNA-Mediated Gene Editing with Nanoparticle Delivery

Scientists report that triplex-forming peptide nucleic acids (PNAs) substituted at the γ position plus stimulation of the stem cell factor/c-Kit pathway yielded high levels of gene editing in hematopoietic stem cells in a mouse model of human β-thalassemia. [Nat Commun] Full Article | Press Release

Bioengineering of Injectable Encapsulated Aggregates of Pluripotent Stem Cells for Therapy of Myocardial Infarction

Researchers showed that pluripotent stem cell aggregates pre-differentiated into the early cardiac lineage and encapsulated in a biocompatible and biodegradable micromatrix, are suitable for injectable delivery. This method significantly improved the survival of the injected cells by more than six-fold compared with the conventional practice of injecting single cells, and effectively prevents teratoma formation. [Nat Commun] Full Article

Pre-Vascularization Enhances Therapeutic Effects of Human Mesenchymal Stem Cell Sheets in Full Thickness Skin Wound Repair

In this work, in vitro cultured hMSC cell sheets (HCS) and pre-vascularized hMSC cell sheets (PHCS) were implanted in a rat full thickness skin wound model covered with an autologous split thickness skin graft (STSG). Results demonstrated that the HCS and the PHCS implantations significantly reduced skin contraction and improved cosmetic appearance relative to the STSG control group. [Theranostics] Full Article | Press Release

Highly Efficient Genome Editing of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9

Investigators describe fast, efficient, and cost-effective methods to directly modify the genomes of mouse and human hematopoietic stem/progenitor cells (HSPCs) using the CRISPR/Cas9 system. Using plasmid and virus-free delivery of guide RNAs alone into Cas9-expressing HSPCs or Cas9-guide RNA ribonucleoprotein complexes into wild-type cells, they have achieved extremely efficient gene disruption in primary HSPCs from mouse and human. [Cell Reports] Full Article | Graphical Abstract

A Non-Viral Suicide Gene Delivery System Traversing the Blood Brain Barrier for Non-Invasive Glioma Targeting Treatment

Herpes simplex virus type I thymidine kinase gene in viral vector is a promising strategy against glioblastoma multiforme. However, the biosafety risk restricts its application in clinic. In this work, poly (l-lysine)-grafted polyethylenimine, which combines the high transfection efficiency of polyethylenimine and the good biodegradability of poly (l-lysine), was adopted as the non-viral vector backbone. [J Control Release] Abstract

Mesenchymal Stem Cells with Sirt1 Overexpression Suppress Breast Tumor Growth via Chemokine-Dependent Natural Killer Cells Recruitment

Scientists demonstrated the effect of mesenchymal stem cells (MSCs) with Sirt1 overexpression (MSCs-Sirt1) in mice bearing 4T1 breast cancer and investigated the underlying mechanism. They found that MSCs could accelerate breast tumor growth with promoted proliferation and inhibited apoptosis, whereas MSCs-Sirt1 significantly suppressed tumor growth with proliferation inhibition and apoptosis promotion. [Sci Rep] Full Article

Association of Epstein–Barr Virus Reactivation with the Recovery of CD4/CD8 Double-Negative T Lymphocytes after Haploidentical Hematopoietic Stem Cell Transplantation

Haploidentical hematopoietic stem cell transplantation (haploHSCT) recipients with EBV-DNAemia were compared with a control group without EBV reactivation, with regard to the recoveries of T-cell subpopulations. In contrast to other T-cell subpopulations, the median counts ofCD4−CD8−T cells in recipients with EBV-DNAemia were significantly lower than the control group at a serial time course after transplantation. [Bone Marrow Transplant] Abstract

Non-Viral Delivery of Genome-Editing Nucleases for Gene Therapy

The authors summarize recent advances that have been made on non-viral delivery of genome-editing nucleases. In particular, they focus on non-viral delivery of Cas9/sgRNA ribonucleoproteins for genome editing. Additionally, the future direction for developing non-viral delivery of programmable nucleases for genome editing is discussed. [Gene Ther] Abstract

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

$50,000 in Awards Presented to Innovators by Ontario Centers of Excellence

More than 360 guests participated in Ontario Centers of Excellence’s (OCE’s) AGM and annual awards presentation at Design Exchange in downtown Toronto. In addition to presenting $50,000 in awards, highlights of the evening included the release of OCE’s 2015/16 Annual Report, an on-site showcase of 24 OCE-funded companies representing some of the past year’s great success stories, and an address by the Honourable Reza Moridi, Ontario’s Minister of Research, Innovation and Science. [CNW Group Ltd.] Press Release

Histogenics Corporation Provides NeoCart^® Phase III Clinical Trial Enrollment Update

Histogenics Corporation announced that as of October 24, 2016 it had enrolled 183, or approximately three-quarters, of the 245 patients required to complete enrollment of its ongoing NeoCart Phase III clinical trial. This trial is being conducted under a Special Protocol Assessment with the United States Food and Drug Administration. [Histogenics Corporation] Press Release

Results from Shire’s Phase II Study of Maribavir Showed Activity against CMV Infection in Patients Undergoing Transplant

Shire plc presented data from a Phase II study evaluating maribavir, an investigational antiviral agent studied in patients with cytomegalovirus (CMV) infection undergoing hematopoietic stem cell transplant or solid organ transplant who are resistant or refractory to ganciclovir or foscarnet, drugs currently used to treat these infections. [Shire plc] Press Release

BrainStorm Granted United States Patent for its Stem Cell Technology

BrainStorm Cell Therapeutics Inc. announced that it has been granted United States Patent No. 9,474,787 titled “Mesenchymal Stem Cells for the Treatment of CNS Diseases.” The allowed claims cover mesenchymal stem cells that secrete neurotrophic factors, including brain-derived neurotrophic factor and glial derived neurotrophic factor, as well as a pharmaceutical compositions comprising these factors. [BrainStorm Cell Therapeutics Inc.] Press Release

Biogen’s Regulatory Applications for Nusinersen as a Treatment for Spinal Muscular Atrophy Accepted by FDA and EMA

Biogen announced that its New Drug Application for nusinersen, an investigational treatment for spinal muscular atrophy, has been accepted by the U.S. Food and Drug Administration (FDA) for Priority Review, and that the company’s Marketing Authorization Application has been validated by the European Medicines Agency (EMA). [Biogen] Press Release

Solid Biosciences Granted U.S. and E.U. Orphan Drug Designations for Lead Gene Therapy Candidate for Duchenne Muscular Dystrophy

Solid Biosciences and its subsidiary, Solid GT, announced that the U.S. Food and Drug Administration and the European Commission have granted Orphan Drug designations for the company’s gene therapy candidate, SGT-001, for the treatment of patients with Duchenne muscular dystrophy. Solid plans to initiate clinical studies for SGT-001 in 2017. [Solid Biosciences] Press Release

HemaCare Signs Distribution Agreement with Korea-Based Partner

HemaCare Corporation has expanded its global capabilities through a strategic distribution agreement in Korea with Seoul-based AccuResearch Korea, Inc. This partnership ensures researchers in South Korea can access HemaCare’s human healthy and disease state fresh and cryopreserved hematopoietic cell products for their basic scientific and cell therapy research and development needs in a timely manner and with local regional support. [HemaCare Corporation (Business Wire, Inc.)] Press Release

Two Baylor College of Medicine Faculty Members Named to National Academy of Medicine

Dr. Malcolm Brenner, founding director of the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist Hospital, and Dr. Cheryl Walker, founding director of the Center for Precision Environmental Health at Baylor, both renowned leaders in their respective fields, were elected to membership in the National Academy of Medicine. [Baylor College of Medicine] Press Release

Scientific Challenges Loom for Canada’s Popular Prime Minister

Many in Canada’s science community say they are reserving judgement, waiting to see whether Trudeau can sustain his string of victories as he tackles some of country’s thorniest science-policy issues. [Nature News] Editorial

Review Finds Misconduct in Events Surrounding WHO Fetal Growth Study

For the first time in its 68-year history, the World Health Organization (WHO) has concluded that researchers are guilty of research misconduct. An independent review commissioned by WHO has found that “research ethics misconduct occurred” in a multimillion-dollar global study on fetal growth led by researchers at the University of Oxford in the United Kingdom. [ScienceInsider] Editorial

Two Major California Research Institutes Will Merge

One of the biggest nonprofit biomedical research outfits in the world is getting a new translational medicine research arm, aimed at speeding the conversion of basic research insights into novel medicines. Officials at the Scripps Research Institute announced that it will merge with the California Institute for Biomedical Research, which was launched in 2012 as a nonprofit version of a drug development company. [ScienceInsider] Editorial

To Save Money, NSF Requires University Cost-Sharing for Rotators

The National Science Foundation (NSF) has decided that universities should pay 10% of the salaries of faculty members working temporarily at the agency. NSF hopes the new policy will demonstrate its commitment to saving taxpayer dollars without alienating the academic community that it relies upon to stay on the cutting edge of basic science. [ScienceInsider] Editorial

FDA

Labeling for Permanent Hysteroscopically Placed Tubal Implants Intended for Sterilization; Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc. No:2016-26243) Notice

Authorizations of Emergency Use of In Vitro Diagnostic Devices for Detection and/or Diagnosis of Zika Virus; Availability (FR Doc. No:2016-26066) Notice

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; General Licensing Provisions: Biologics License Application, Changes to an Approved Application, Labeling, Revocation and Suspension, and Postmarketing Studies Status Reports (FR Doc. No:2016-26064) Notice

NEW Global Forum on Transplantation Research and Technologies
November 28-30, 2016
Dubai, United Arab Emirates

Visit our events page to see a complete list of events in the community.

NEW Principal Scientist – Cellular Therapeutics (Celgene Corporation)

NEW Associate Director – Cellular Process Development (Celgene Corporation)

Research Specialist – Tissue Regeneration Research In Vitro and In Vivo (University of Pittsburgh)

Head of Laboratory – Cellular and Molecular Therapies (NHS Blood and Transplant)

Postdoctoral Fellow – Anti-Tumor Immunity in Tetraspannin Deficient Mice (Radboud University Medical Center)

Postdoctoral Fellowship – Genetic Medicine (Weill Cornell Medical College)

Research Fellow in Pluripotent Stem Cells Self-Renewal Program and Cell Cycle (KU Leuven)

Faculty Position – Stem Cell Biology and Regenerative Medicine (Stanford University)

Research Associate – Hematology (Editas Medicine)

Faculty Position – Stem Cell Research (Children’s Research Institute at UTSW)

Assistant Professorship – Stem Cells and Regenerative Medicine (University of Southern California)

Postdoctoral Positions – Stem Cell Research, Hematopoiesis, Cancer, Genome Editing (Icahn School of Medicine)

Faculty Position – Cancer Research (Children’s Research Institute at UTSW)

Assistant Professor – Molecular and Cellular Biology (Harvard University)

Postdoctoral Fellow – Novel Systems for Treatment of Type 1 Diabetes (Toronto General Research Institute & McEwen Centre for Regenerative Medicine)

Assistant Professor – Developmental Biology (Florida State University)

Research Associate – Cellular Therapy (California Institute for Biomedical Research)

Postdoctoral Fellow – Pluripotent Stem Cell Technology (University of California Davis)

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

Postdoctoral Fellow – Pathogenic Mechanisms of Rare Bone Disorders (University of Connecticut Health)

Postdoctoral Fellow – Oxidative Lung Injury (Temple University)

Postdoctoral Fellow – Lentivirus-Mediated FOXP3 Gene Transfer (Stanford University School of Medicine)

Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.

Have we missed an important article or publication in Cell Therapy News? Click here to submit!

Comments or suggestions? Submit your feedback here.