Cell Therapy News Volume 17.40 | Nov 7 2016

TOP STORY

CRISPR/Cas9 β-Globin Gene Targeting in Human Hematopoietic Stem Cells

Scientists present a CRISPR/Cas9 gene-editing system that combines Cas9 ribonucleoproteins and adeno-associated viral vector delivery of a homologous donor to achieve homologous recombination at the β-globin (HBB) gene in hematopoietic stem cells. Notably, they devised an enrichment model to purify a population of hematopoietic stem and progenitor cells with more than 90% targeted integration. [Nature] Abstract | Press Release

(Ranked by impact factor of the journal)

Cardiac Repair in Guinea Pigs with Human Engineered Heart Tissue from Induced Pluripotent Stem Cells

Investigators created human engineered heart tissue (hEHT) strips from human induced pluripotent stem cell (hiPSC)–derived cardiomyocytes and hiPSC-derived endothelial cells. The hEHTs were transplanted onto large defects of guinea pig hearts seven days after cryoinjury, and the results were compared with those obtained with human endothelial cell patches or cell-free patches. [Sci Transl Med] Abstract

Matching for the MICA-129 Polymorphism Is Beneficial in Unrelated Hematopoietic Stem Cell Transplantation

Researchers investigated the effect of MICA (MHC class I polypeptide-related sequence A)-matching on survival after unrelated hematopoietic stem cell transplantation. They sequenced 2172 patients and their respective donors for MICA. [Blood] Abstract

Multiplex Genome Editing to Generate Universal CAR T Cells Resistant to PD1 Inhibition

By combining the lentiviral delivery of chimeric antigen receptor (CAR) and CRISPR RNA electroporation to co-introduce RNA encoding the Cas9 and gRNAs targeting endogenous TCR, beta-2 microglobulin and PD1 simultaneously, to generate gene-disrupted allogeneic CAR T cells deficient of TCR, HLA class I molecule and PD1. [Clin Cancer Res] Abstract

Neurotensin-Conjugated Reduced Graphene Oxide with Multi-Stage Near-Infrared-Triggered Synergic Targeted Neuron Gene Transfection In Vitro and In Vivo for Neurodegenerative Disease Therapy

Neurotensin -conjugated polyethylenimine -modified reduced graphene oxide nanoparticles with precisely controlled two-stage near-infrared -laser photothermal treatment to enhance the ability to target neurons and achieve high gene transfection in neurons. [Adv Healthc Mater] Abstract

Overexpression of Soluble RAGE in Mesenchymal Stem Cells Enhances their Immunoregulatory Potential for Cellular Therapy in Autoimmune Arthritis

Researchers determined whether soluble receptor for advanced glycation end products (sRAGE) has therapeutic effects and the mechanisms active in sRAGE-overexpressing mesenchymal stem cells (sRAGE-MSCs) in an experimental model of rheumatoid arthritis. sRAGE-MSCs were generated by DNA transfection of human adipose tissue-derived MSCs. [Sci Rep] Full Article

Bioinspired Seeding of Biomaterials Using Three Dimensional Microtissues Induces Chondrogenic Stem Cell Differentiation and Cartilage Formation under Growth Factor Free Conditions

The authors integrated the microenvironment of cellular condensation into biomaterials by encapsulating microaggregates of a hundred human periosteum-derived stem cells. This resulted in decreased stemness-related markers, up regulation of chondrogenic genes and improved in vivo cartilage tissue formation, as compared to single cell seeded biomaterials. [Sci Rep] Full Article

Transplantation of Adipose Tissue Derived Mesenchymal Stem Cell (ATMSC) Expressing Alpha-1 Antitrypsin Reduces Bone Loss in Ovariectomized Osteoporosis Mice

Eight-week-old female C57BL6 mice underwent ovariectomy and treated with human AAT (hAAT), ATMSC, ATMSC+hAAT, ATMSCs infected with lentiviral vectors expressing hAAT. Scientists showed that lenti-hAAT vector infected ATMSCs produced high levels of hAAT. [Hum Gene Ther] Abstract

Tacrolimus Plus Sirolimus with or without ATG as GVHD Prophylaxis in HLA-Mismatched Unrelated Donor Allogeneic Stem Cell Transplantation

Researchers performed a multicenter, retrospective study comparing tacrolimus and sirolimus with or without antithymocyte globulin (ATG) in 104 patients who underwent mismatched unrelated donors hematopoietic stem cell transplantation. [Bone Marrow Transplant] Abstract

Evidence for CD19B–CD8T Cell Interactions in Blood and Tissues from Patients with GvHD

The authors described an interaction between autologous CD19 B cells and activated CD8 cytotoxic T cells (CTLs) in normal donors. In vitro Ag-stimulated CTLs form stable couplets with B cells independent of Ag presentation, through CD27 (on B cells)–CD70 (on T cells) axis. [Bone Marrow Transplant] Full Article

Dental Pulp Stem Cells Express Tendon Markers under Mechanical Loading and Are a Potential Cell Source for Tissue Engineering of Tendon-Like Tissue

Investigators explored the possibility of dental pulp stem cells for potential application in tendon tissue engineering. The expression of tendon-related markers such as scleraxis, tenascin-C, tenomodulin, eye absent homologue 2, collagens I and VI was detected in dental pulp tissue. [Int J Oral Sci] Full Article

Injectable Biomaterials for Stem Cell Delivery and Tissue Regeneration

The authors focused on injectable cell carriers which are not only expected to improve therapeutic outcomes, but also to facilitate easy surgical process. Such cell carriers include in situ gelling hydrogel, injectable supramolecular hydrogels, and microcarriers. The current design of hydrogels and microcarriers can achieve biocompatibility, biodegradability, and provide desirable features to enhance biological response. [Expert Opin Biol Ther] Abstract

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

Axiogenesis and Metrion Biosciences Announce Stem Cell Collaboration

Axiogenesis AG and Metrion Biosciences Ltd have signed a collaboration agreement to validate, optimize and commercialize induced pluripotent stem cell-derived cell based assays to facilitate drug discovery and more accurately predict cardiac arrhythmia and neuronal risk. [Axiogenesis AG] Press Release

Sernova and CTI Clinical Trial Announce Regulatory Collaboration

Sernova Corp. announced their collaboration on regulatory matters respecting Sernova’s Cell Pouch System, a novel implantable and scalable medical device that when combined with therapeutic cells, may provide a convenient, safe and effective long-term therapeutic option for patients with chronic diseases such as insulin-dependent diabetes who seek to improve their quality of life. [Sernova Corp.] Press Release

TissueGene Licensee, Kolon Life Science, Partners with Mitsubishi Tanabe Pharma to Develop and Commercialize Invossa^™, the World’s First Cell-Mediated Gene Therapy for Degenerative Osteoarthritis

TissueGene, Inc. announced an exclusive licensing and development agreement between Mitsubishi Tanabe Pharma Corporation of Japan and TissueGene’s Asia licensee, Kolon Life Science of Korea. The agreement between the two companies focuses on the development and commercialization of Invossa^™, the world’s first cell-mediated gene therapy for degenerative osteoarthritis for the Japanese market. [TissueGene, Inc.] Press Release

University of California Irvine (UCI) Doses First Patient in Phase IIa Clinical Trial Using Stemedica’s Stem Cells for Alzheimer’s Disease

Stemedica International, SA announced enrollment of its first patient at UCI. The study, is entitled “A Phase IIa Multicenter, Randomized, Single-blind, Placebo-controlled, Crossover Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Allogeneic Human Mesenchymal Stem Cells in Subjects with Mild to Moderate Dementia Due to Alzheimer’s disease.” [Stemedica International (PR Newswire Association LLC.)] Press Release

Renova Therapeutics Announces Successful End-of-Phase II Meeting with FDA for RT-100 AC6 Gene Transfer Therapy

Renova^™ Therapeutics announced that it has completed an End-of-Phase II meeting with the U.S. Food and Drug Administration (FDA) for its lead gene therapy product candidate, RT-100 AC6 gene transfer for the treatment of patients with heart failure and reduced ejection fraction. [TissueGene, Inc.] Press Release

LifeNet Health Expands Regenerative Medicine Efforts with Opening of Research Triangle Park, N.C., Facility

LifeNet Health is expanding its cell-based research and processing capacity with the opening of a facility in North Carolina’s Research Triangle Park, near Durham. It will be an extension of its flagship research center, the LifeNet Health Institute of Regenerative Medicine, located at the company’s headquarters in Virginia Beach, Va. [LifeNet Health] Press Release

LifeNet Health Announces New Advanced Technology Platforms with Acquisition of Vivo Biosciences, Inc., Assets

LifeNet Health has acquired the assets of Alabama-based biotechnology company Vivo Biosciences, Inc., as part of the organization’s growing emphasis on applying cell-based innovations to the process of pharmaceutical discovery and personalized medicine. [LifeNet Health] Press Release

Regenicin, Inc. Completes Successful Pre-IND Meeting with Food and Drug Administration (FDA) for its New Autologous Cultured Skin Substitute, NovaDerm^®

Regenicin, Inc. announced that they have completed the Pre-IND (Investigational New Drug) Meeting via teleconference with members of the FDA Office of Cellular, Tissue, and Gene Therapies in the Center for Biologics Evaluation and Research regarding its product NovaDerm^®. [Regenicin, Inc. (PR Newswire Association LLC.)] Press Release

Creative Medical Technologies Receives Exclusive License to Key Amniotic Fluid Stem Cell Patent from University of California System

Creative Medical Technology Holdings, Inc. announced signing of an exclusive license agreement with the University of California System to commercialize and develop a novel type of amniotic fluid derived stem cell as disclosed in issued US Patent No. 7,569,385. [Creative Medical Technology Holdings, Inc. (PR Newswire Association LLC.)] Press Release

Hard Work, Little Reward: Nature Readers Reveal Working Hours and Research Challenges

A poll answered by thousands of Nature readers suggests that nearly two-thirds have considered quitting research, and that almost 40% work more than 60 hours in a week. The fight for funding is the greatest challenge facing early-career scientists, readers said. [Nature] Editorial

Twitterstorm Shows Why Scientific Evidence Matters

Grammar and Twitter rarely sit happily, so it would be churlish to point out that when the Welsh MP Glyn Davies tweeted at the weekend: “Nothing more irritating than academics ­rubbishing the efforts of those operating at the sharp end, without facing up to the hard decisions”, he was inadvertently complaining that people at the sharp end (himself included presumably) do not confront hard decisions. [Nature] Editorial

FDA

Agency Information Collection Activities; Proposed Collection; Comment Request; Human Tissue Intended for Transplantation (FR Doc. No:2016-26398) Notice

Authorizations of Emergency Use of In Vitro Diagnostic Devices for Detection of Zika Virus; Availability (FR Doc. No:2016-26532) Notice

Agency Information Collection Activities; Proposed Collection; Comment Request; Guidance for Industry: Use of Serological Tests To Reduce the Risk of Transmission of Trypanosoma cruzi Infection in Whole Blood and Blood Components Intended for Transfusion (FR Doc. No:2016-26792) Notice

NEW Keystone Symposium: TGFβ in Immunity, Inflammation and Cancer
January 9-13, 2017
Taos, United States

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

NEW Assistant or Associate Member – Stem CellGene Therapy (Fred Hutchinson Cancer Research Center)

NEW Medical Director – Immuno-Oncology with T Cell Therapies (Celgene Corporation)

Principal Scientist – Cellular Therapeutics (Celgene Corporation)

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 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)

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

Postdoctoral Fellow – Oxidative Lung Injury (Temple University)

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.