Cell Therapy News Volume 18.28 | Aug 21 2017

TOP STORY

Engraftment and In Vivo Proliferation Advantage of Gene Corrected Mobilized CD34⁺ Cells from Fanconi Anemia Patients

The authors demonstrated that a validated short transduction protocol of G-CSF plus plerixafor-mobilized CD34⁺ cells from Fanconi anemia (FA)-A patients with a therapeutic FANCA-lentiviral vector corrects the phenotype of in vitro cultured hematopoietic progenitor cells. Transplantation of transduced FA CD34⁺ cells into immunodeficient mice resulted in reproducible engraftment of myeloid, lymphoid and CD34⁺ cells. [Blood] Abstract

(Ranked by impact factor of the journal)

Neural Crest Cell Implantation Restores Enteric Nervous System Function and Alters the Gastrointestinal Transcriptome in Human Tissue-Engineered Small Intestine

Human pluripotent stem cell-derived enteric neural crest cell (ENCC) supplementation of human intestinal organoid (HIO)-tissue-engineered small intestine (TESI) established submucosal and myenteric ganglia, repopulated various subclasses of neurons, and restored neuroepithelial connections and neuron-dependent contractility and relaxation in ENCC-HIO-TESI. [Stem Cell Reports] Full Article | Graphical Abstract

Mitigating Ischemic Injury of Stem Cell-Derived Insulin-Producing Cells after Transplant

Investigators showed that more than half of stem cell-derived insulin-producing cells (SCIPCs) die shortly after transplantation. A two-pronged strategy of physiological oxygen acclimatization during differentiation and amino acid supplementation during transplantation significantly improved SCIPC survival after transplant. [Stem Cell Reports] Full Article | Graphical Abstract

Administration of Mesenchymal Stromal Cells before Renal Ischemia/Reperfusion Attenuates Kidney Injury and May Modulate Renal Lipid Metabolism in Rats

Researchers compared the impact of mesenchymal stromal cell injection seven days before versus one day after renal ischemia/reperfusion in rats. Control groups received equivalent volumes of saline at similar time-points. [Sci Rep] Full Article

CRISPR/Cas9-Mediated Deletion of Foxn1 in NOD/SCID/IL2rg^−/− Mice Results in Severe Immunodeficiency

The authors generated a novel strain of nude NOD/SCID/IL2rg^−/− mice by knocking out Foxn1 from NOD/SCID/IL2rg^−/− mice using the CRISPR/Cas9 system. [Sci Rep] Full Article

Improved Hematopoietic Gene Therapy in a Mouse Model of Fanconi Anemia Mediated by Mesenchymal Stromal Cells

Because the engraftment of corrected hematopoietic stem cells is particularly challenging in Fanconi anemia (FA), scientists investigated the relevance of mesenchymal stromal cells in an experimental model of FA gene therapy. [Hum Gene Ther] Abstract

Therapy with Mesenchymal Stromal Cells or Conditioned Medium Reverse Cardiac Alterations in a High-Fat Diet–Induced Obesity Model

Researchers evaluated the therapeutic potential of mesenchymal stromal cells and their conditioned medium to treat cardiac complications in a mouse model of high-fat diet–induced obesity. [Cytotherapy] Abstract

Repeated AAV-Mediated Gene Transfer by Serotype Switching Enables Long-Lasting Therapeutic Levels of hUgt1a1 Enzyme in a Mouse Model of Crigler-Najjar Syndrome Type I

To establish an effective and long lasting cure, the authors applied adeno-associated virus (AAV)-mediated liver gene therapy to a relevant mouse model of the disease. Repeated gene transfer to adults by AAV-serotype switching, upon neonatal administration, resulted in life-long correction of total bilirubin levels in both genders. [Gene Ther] Abstract

Term vs. Preterm Cord Blood Cells for the Prevention of Preterm Brain Injury

Allogeneic umbilical cord blood cells from term or preterm sheep, or saline, were administered to the fetus at 12 hours after hypoxia–ischemia (HI). The fetal brain was collected at ten-day post HI for assessment of white matter neuropathology. [Pediatr Res] Abstract

Regenerative Medicine and Cell-Based Approaches to Restore Pancreatic Function

Scientists describe progress and challenges in cell-based methods to restore pancreatic function, with a focus on optimizing the site for cell delivery and decreasing requirements for immunosuppression through encapsulation. They also discuss the tools and strategies being used to generate exocrine pancreas and insulin-producing β-cell surrogates in situ and highlight obstacles to clinical application. [Nat Rev Gastroenterol Hepatol] Abstract

Advances and Challenges in Cardiovascular Gene Therapy

The authors focus on the current status of cardiovascular gene therapy with an emphasis on myocardial gene therapy targets and trials. They emphasize the value of bidirectional translationality from bench to bedside and back, and suggest novel approaches to better fulfill the clinical demand for novel therapies. [Hum Gene Ther] Abstract

Gene Delivery Approaches for Mesenchymal Stem Cell Therapy: Strategies to Increase Efficiency and Specificity

Investigators review current gene delivery approaches, including viral and plasmid vectors, for transfecting mesenchymal stem cells with beneficial genes. They also discuss the use of a few emerging technologies that could be used to improve the transfer/induction of desirable genes for cell therapy. [Stem Cell Rev] Abstract

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

Blueprint Medicines to Present Updated Data from Ongoing Phase I Clinical Trial of BLU-554 in Patients with Advanced Hepatocellular Carcinoma

Blueprint Medicines Corporation announced that updated data from its ongoing Phase I clinical trial evaluating BLU-554 in patients with advanced hepatocellular carcinoma will be presented in oral presentations. [Press release from Blueprint Medicines Corporation discussing research to be presented at the European Society for Medical Oncology (ESMO) 2017 Congress, Madrid] Press Release

BioLineRx Announces Regulatory Submission of Phase III Registrational Study for BL-8040 in Stem Cell Mobilization

BioLineRx Ltd. announced the filing of regulatory submissions required to commence a randomized, controlled Phase III registrational trial of BL-8040 for the mobilization of hematopoietic stem cells for autologous transplantation in patients with multiple myeloma. [BioLineRx Ltd.] Press Release

Gamida Cell Announces First Patient Transplanted in Phase I/II Study of CordIn for Severe Aplastic Anemia and Hypoplastic MDS

The first subject has been transplanted in an investigator-initiated study of Cordin™ for patients with severe aplastic anemia or hypoplastic myelodysplastic syndrome (MDS) who have no available matched donor. [Gamida Cell] Press Release

Cellectis’ UCART123 Administered to First Patient with BPDCN in Phase I Clinical Trial at MD Anderson Cancer Center

Cellectis announced that the first patient with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) has been dosed in Cellectis’ Phase I clinical study using the company’s wholly controlled TALEN^® gene edited product candidate UCART123 at the MD Anderson Cancer Center. [Cellectis] Press Release

Applied StemCell Awarded $1.3 Million USD from NIH for TARGATT™ Gene Editing

Applied StemCell, Inc. was awarded close to $1.3 million US dollars in a small Business Innovation Research grant from the NIH. [Applied StemCell, Inc.] Press Release

Abeona Therapeutics Announces Pivotal Expansion of Gene Therapy Trials in Sanfilippo Syndrome Type A

Abeona Therapeutics Inc. announced the pivotal expansion of its gene therapy clinical trials for patients with mucopolysaccharidosis type IIIA in the USA, Europe and Australia. [Abeona Therapeutics Inc.] Press Release

U.S. Department of Defense to Conduct Studies of Pluristem’s PLX-R18 in a New ARS Project for Use before Radiation Exposure

Pluristem Therapeutics Inc. announced that a pilot study of the company’s PLX-R18 cell therapy will be initiated by the U.S. Department of Defense’s Armed Forces Radiobiology Research Institute, part of the Uniformed Services University of Health Sciences. [Pluristem Therapeutics Inc.] Press Release

RepliCel Life Sciences Joins MedCision’s ThawSTAR^® Early Adopter Program

MedCision, LLC and RepliCel Life Sciences, Inc. announced that RepliCel has joined MedCision’s ThawSTAR^® Early Adopter Program, a novel program that helps companies and investigators de-risk thawing of cell therapies in early stage clinical trials through to commercialization and point-of-care. [RepliCel Life Sciences, Inc.] Press Release

ACF Bioservices Completes cGMP-Compliant Testing Facility for Gene and Cell Therapy Products

ACF Bioservices™ announced the completion of construction on its dedicated cGMP-compliant 3400 square foot facility for gene and cell therapy product testing. [ACF Bioservices™ (PR Newswire Association LLC.)] Press Release

NSF Reiterates Policy on Teaching Good Research Habits despite Its Limitations

The National Science Foundation (NSF) in Arlington, Virginia, has decided to double down on its implementation of a congressionally mandated policy aimed at reducing research misconduct among NSF-funded scientists, despite a new report that notes problems with the agency’s approach. [ScienceInsider] Editorial

Trump’s First List of Science Priorities Ignores Climate—and Departs from His Own Budget Request

President Donald Trump has translated his campaign promise to “make America great again” into his administration’s first blueprint for federal investment in science and technology. [ScienceInsider] Editorial

China’s Embrace of Embryo Selection Raises Thorny Questions

Fertility centers are making a massive push to increase preimplantation genetic diagnosis in a bid to eradicate certain diseases. [Nature News] Editorial

Budget Cuts Fuel Frustration among Japan’s Academics

Japan’s premier scientific research institution, RIKEN, turned 100 this year, and celebrated with a grand ceremony attended by the empress and emperor. But not everybody was in the mood to party. In the old days, RIKEN was known as a paradise for scientists because of its generous funding. No longer: as Japan cuts off funds in the face of continuing financial uncertainty, the cracks are starting to show. [Nature News] Editorial

NIH

Government-Owned Inventions; Availability for Licensing (FR Doc. No:2017-17438) Notice

Submission for OMB Review; 30-Day Comment Request Chimpanzee Research Use Form (Office of the Director) (FR Doc. No:2017-17411) Notice

NEW Cell Symposia: CRISPR: From Biology to Technology and Novel Therapeutics
October 22-24, 2017
Sitges, Spain

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

NEW Postdoctoral Fellow – Cell Biology, Tumor Immunology and Targeted Therapy (University of Minnesota)

Postdoctoral Associate – Enhancer RNA Therapy (Sylvester Comprehensive Cancer Center)

Postdoctoral Fellow – Cell and Gene Therapy Studies Targeting Heart Failure (Icahn School of Medicine at Mount Sinai)

Research Group Leaders (Centro de Investigación Príncipe Felipe)

Postdoctoral Research Associate – Molecular Mechanisms of Stem Cells in the Heart (University of Louisville)

Research Associate – Cell Engineering (Caribou Biosciences, Inc.)

Quality Improvement Coordinator – Cellular Therapy Core (University of Texas Health Science Center at Houston)

Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

Postdoctoral Fellow – Various Projects (University of Oklahoma)

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