Cell Therapy News 18.13 April 17, 2017 | |
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TOP STORYConversion of Brain Cells Offers Hope for Parkinson’s Patients Researchers have made significant progress in the search for new treatments for Parkinson’s disease. By manipulating the gene expression of non-neuronal cells in the brain, they were able to produce new dopamine neurons. [Press release from Karolinska Institutet discussing online prepublication in Nature Biotechnology] Press Release | Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)In Situ Programming of Leukemia-Specific T Cells Using Synthetic DNA Nanocarriers Scientists demonstrated that DNA-carrying nanoparticles can efficiently introduce leukemia-targeting chimeric antigen receptor genes into T-cell nuclei, thereby bringing about long-term disease remission. [Nat Nanotechnol] Abstract Researchers used a personalized immunogenomic approach to elucidate the global landscape of antitumor T cell responses in complete regression of human papillomavirus–associated metastatic cervical cancer after tumor-infiltrating adoptive T cell therapy. [Science] Abstract To assess the in vivo leukemogenic activity of constitutively active ABL and ARG, investigators generated a bone marrow transplantation model using the chimeric forms TEL/ABL and TEL/ARG, which have comparable kinase activities. [Leukemia] Abstract PBX3 Is Essential for Leukemia Stem Cell Maintenance in MLL-Rearranged Leukemia To examine the role of Pbx3 in acute myeloid leukemia (AML) maintenance and progression, researchers used the CRISPR/Cas9 system to delete Pbx3 in leukemic cells in the MLL-AF9 induced AML mouse model. They found that Pbx3 deletion significantly prolonged the survival of leukemic mice and decreased the leukemia burden by decreasing the capacity of leukemia stem cells (LSCs) and promoting LSC apoptosis. [Int J Cancer] Abstract The authors aimed to determine effects of transplanted unmodified human bone marrow CD34+ (hBM34+) cells into symptomatic G93A mice towards blood-spinal cord barrier repair. Thirteen weeks old G93A mice intravenously received one of three different doses of hBM34+ cells. [Sci Rep] Full Article Researchers found that activin B was able to promote adipose-derived stem cell (ADSC) migration by inducing actin stress fiber formation in vitro. In vivo, activin B in combination with ADSCs was capable of enhancing α-SMA expression and wound closure. [Int J Biochem Cell Biol] Abstract CRISPR-Cpf1 Correction of Muscular Dystrophy Mutations in Human Cardiomyocytes and Mice Investigators deployed Cpf1, a unique class 2 CRISPR (clustered regularly interspaced short palindromic repeats) effector, to correct X-linked dystrophin gene (DMD) mutations in patient-derived induced pluripotent stem cells and mdx mice, an animal model of Duchenne muscular dystrophy. [Sci Adv] Full Article | Press Release To assess the role that host genetic variation has on efficacy of gene therapy, the authors characterized lentiviral-vector gene therapy within a set of 12 collaborative cross mouse strains. Lentiviral vectors carrying the firefly luciferase cDNA under the control of a liver-specific promoter were administered to female mice, with total-body and hepatic luciferase expression periodically monitored through 41 weeks post-vector administration. [Mol Ther Methods Clin Dev] Full Article | |
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REVIEWSAdipose-Derived Stem Cell Therapies for Bone Regeneration Adipose-derived stem cells offer numerous advantages over other stem cells in terms of feasibility of clinical translation. Data obtained from in vivo experimentation are encouraging, and clinical trials are ongoing. More robust validations are thus expected to be achieved during the next few years, and will likely pave the way to optimized patient-tailored treatments for bone regeneration. [Expert Opin Biol Ther] Abstract Advances in Umbilical Cord Blood Cell Therapy: The Present and the Future The authors cover the advances in umbilical cord blood transplantation (UCBT) to overcome the limitation regarding cellular dose, immunological naivety and additional cell doses such as DLI. They also provide an overview regarding the progress in adoptive cellular therapy using umbilical cord blood. [Expert Opin Biol Ther] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSArrowhead Pharmaceuticals Announces Presentations at The International Liver Congress™ Arrowhead Pharmaceuticals Inc. announced that it will present clinical data on ARC-520, ARC-521, and ARC-AAT, the company’s prior generation investigational medicines that were being studied for the treatment of chronic hepatitis B infection and liver disease associated with alpha-1 antitrypsin deficiency. [Press release from Arrowhead Pharmaceuticals Inc. discussing research to be presented at The International Liver Congress™ 2017 (ILC), Amsterdam] Press Release | |
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INDUSTRY NEWSThe Human Vaccines Project, Vanderbilt and Illumina Join Forces to Decode the Human Immunome The Human Vaccines Project and Vanderbilt University Medical Center announced that they joined forces with Illumina, Inc., to decipher the human immunome, the genetic underpinnings of the immune system. Illumina will provide the genetic sequencing technologies and expertise required to process the massive amounts of data required to decode the human immunome. [The Human Vaccines Project (PR Newswire Association, LLC.)] Press Release Medicenna Treats First Patient in Phase IIb Recurrent Glioblastoma Trial Medicenna BioPharma Inc. announced that it has treated the first patient in its Phase IIb clinical trial of MDNA55, the company’s lead candidate. [Medicenna BioPharma Inc.] Press Release Oncolytics Biotech® Inc. Announces Registration Pathway and Clinical Development Plan Oncolytics Biotech® Inc. announced its initial registration pathway and clinical development plan for REOLYSIN®, its proprietary immuno-oncology viral agent. [Oncolytics Biotech® Inc.] Press Release Plasticell announced it has secured Biomedical Catalyst funding of over £920,000 from Innovate UK to create a safe, effective red blood cell substitute for human clinical transfusion, in collaboration with stem cell scientists at the University of Edinburgh. [Plasticell] Press Release Keystone Nano Announces Progress On RNA Delivery Keystone Nano, Inc. (KN) announced significant progress on the delivery of RNA using NanoJackets. KN’s NanoJackets are proprietary, customizable calcium phosphate nanoparticles that protect the active agent in the bloodstream and deliver it to targeted cells. [Keystone Nano, Inc. (PR Newswire Association, LLC.)] Press Release Clinician–Researcher to Lead New Bone Marrow Transplantation Initiative Hematologist–oncologist Ahmad Samer Al-Homsi, MD, MBA, will lead a new bone marrow transplantation program at NYU Langone’s Perlmutter Cancer Center for treating blood-borne cancers, including leukemia, lymphoma, and multiple myeloma, and potentially utilize transplantation as an adjunct to immunotherapy for solid tumors. [NYU Hospitals Center] Press Release | |
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POLICY NEWSWith This New System, Scientists Never Have to Write a Grant Application Again Almost every scientist agrees: Applying for research funding is a drag. Writing a good proposal can take months, and the chances of getting funded are often slim. Funding agencies, meanwhile, spend more and more time and money reviewing growing stacks of applications. That’s why two researchers are proposing a radically different system that would do away with applications and reviews; instead scientists would just give each other money. [ScienceInsider] Editorial Joining the European Union Leads to Less Cross-Border Collaboration When the European Union expanded in the 2000s, it had a negative impact on scientific collaborations between researchers in new member countries and elsewhere, a study has found. [Nature News] Editorial U.S. Report Calls for Research Integrity Board A new report from the National Academies of Sciences, Engineering, and Medicine entitled Fostering Integrity in Research recommends that universities and scientific societies create, operate, and fund a new, independent, nongovernmental Research Integrity Advisory Board. [ScienceInsider] Editorial Billion-Dollar Boost Sought for Canadian Science The Canadian government should increase its support for fundamental science by more than a third, from Can $3.5 billion per year to Can $4.8 billion, according to a long-awaited independent review of the country’s research priorities and funding. Although Canada’s three major granting agencies and a research-support fund received a record-breaking Can $95-million boost in 2016, their funding stayed flat in the 2017 budget that the government released last month. The science review recommends increases for those three agencies, as well as a handful of other programmes, that would amount to Can $325 million per year for four years. [Nature News] Editorial
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REGULATORYFDARegulation of Intentionally Altered Genomic DNA in Animals; Extension of Comment Period (FR Doc. No:2017-07470) Notice
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EVENTSNEW 2nd Bioprocessing of Advanced Cellular Therapies Congress Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Program Director – CART Cell (Servier) Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic) Executive Director – Global CAR-T Supply (Celgene Corporation) Postdoctoral Associate – Gene Therapy (University of Massachusetts Medical School) Postdoctoral Research Fellow – Preclinical Models of Cancer Immunotherapy (Providence Cancer Center) Postdoctoral Fellow – Various Projects (University of Oklahoma) GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center) Assistant or Associate Member – Stem CellGene Therapy (Fred Hutchinson Cancer Research Center) Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.
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