Cell Therapy News 20.39 November 25, 2019 | |
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TOP STORYThe authors provide a detailed protocol for the design, cloning, delivery, and utilization of such artificial microRNA clusters for gene therapy purposes. The strategy effectively decreased the requirements for molecular cloning, because the nucleic acid sequence encoding the combination of the desired microRNAs was designed and validated in silico and then directly synthesized as DNA that was ready for subcloning into appropriate delivery vectors, for both in vitro and in vivo use. [Nat Protoc] Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)CAR.CD19-NK cells displayed higher antileukemic activity toward CD19+ cell lines and primary blasts obtained from patients with B-cell precursor ALL compared with unmodified NK cells. In vivo animal model data showed that the antileukemia activity of CAR.CD19-NK cell was superimposable to that of CAR-T cells, with a lower xenograft toxicity profile. [Leukemia] Abstract The authors created universal CAR T cells resistant to PD-1 inhibition through multiplexed gene disruption of endogenous T-cell receptor, beta-2 microglobulin and P-1. Triple gene-edited CAR T cells demonstrated enhanced activity in preclinical glioma models. Prolonged survival in mice bearing intracranial tumors was achieved after intracerebral, but not intravenous administration. [J Immunother Cancer] Full Article Effective CRISPR Interference of an Endogenous Gene via a Single Transgene in Mice Scientists tested the effectiveness of a single CRISPRi transgene broadly expressing a single guide RNA and a catalytically dead Cas9 fused to the KRAB repressor domain to suppress a well-characterized target gene, Tnfsf11. The phenotype of CRISPRi transgenic mice was compared to mice with germline deletion of Tnfsf11, which were osteopetrotic and did form lymph nodes. [Sci Rep] Full Article Researchers tested if mutation of the identified candidate genes could cooperate with KrasG12D to generate primary sarcomas in mice. In addition to identifying the well-known tumor suppressor cyclin dependent kinase inhibitor 2A, whose alternative reading frame product p19 activates transformation-related protein 5, they also identified other putative tumor suppressors, such as F-box/WD repeat-containing protein 7 and solute carrier family 9 member 3. [Sci Rep] Full Article Scientists evaluated whether the correction of adipose tissue dysfunction could improve hippocampal synaptic transmission in adipocyte insulin resistance mice vs. their wildtype littermates. Animals were simply randomized to receive mesenchymal cells, then weighed weekly for twelve weeks. [J Neurochem] Abstract Potential of Treated Dentin Matrix Xenograft for Dentin-Pulp Tissue Engineering The expression of dentin matrix acidic phosphoprotein 1, dentin sialophosphoprotein, and osteopontin was evaluated in dental pulp stem cells using quantitative real-time polymerase chain reaction. The samples were then implanted intramuscularly in rats for 30 days, and the inflammatory cells were quantified histologically. [J Endod] Abstract Chondrogenic Differentiation of Human ASCs by Stiffness Control in 3D Fibrin Hydrogel Human adipose-derived stem cells within high-concentration fibrinogen formulation maintained a round morphology similar to natural chondrocytes. Variations in thrombin concentration had a lesser effect on cell morphology. In terms of in vivo cartilage formation, the formulation with 30mg/mL fibrinogen and 100 IU/mL thrombin showed the highest cartilage formation, as evidenced through collagen type II alpha 1 chain and safranin-O, four weeks after implantation. [Biochem Biophys Res Commun] Abstract Compared to wild-type (WT) mice, physiological anatomy indicated that the morphological characters of vital organs in fibroblast growth factor 21 (Fgf21) knock-out (KO) mice were normal.Depilation experiments demonstrated that compared to the WT mice, the hair regrowth speed was reduced in the Fgf21 KO mice. The number of hair shafts in these mice considerably decreased, as indicated by the tissue sample analyses. [Gene] Abstract Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSRational Designs of In Vivo CRISPR-Cas Delivery Systems The authors summarize the features of plasmid-, RNA- and ribonucleoprotein (RNP)-based CRISPR-Cas therapeutics. Then, they survey the current in vivo delivery systems. Finally, they specify the requirements for efficient in vivo delivery in clinical settings, and highlight both efficiency and safety for different CRISPR-Cas tools. [Adv Drug Deliv Rev] Full Article Chimeric Antigen Receptor T Cell Therapies for Multiple Myeloma Investigators summarize recent developments of CAR-T therapy in multiple myeloma, focusing on promising targets, new technologies, and new research areas. Additionally, a comprehensive overview of antigen selection is presented along with preliminary results and future directions of CAR-T therapy development. [J Hematol Oncol] Full Article Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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INDUSTRY NEWSCRISPR Therapeutics and Vertex Pharmaceuticals Incorporated announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase I/II clinical trials. [CRISPR Therapeutics] Press Release Ohio State-Developed Drug Receives FDA Approval for Treatment of CLL and SCL The FDA approved the use of the drug acalabrutinib for first-line therapy in chronic lymphocytic leukemia (CLL) and small cell lymphoma (SCL). This is the first full approval of the targeted drug therapy, which was developed and tested at The Ohio State University Comprehensive Cancer Center- Arthur G. James Cancer Hospital and Richard J. Solove Research Institute in collaboration with the pharmaceutical partner, Acerta Pharma. [Ohio State University] Press Release An innovative new major private-public partnership led by Harvard and MIT aims to accelerate one of the hottest and most promising areas of medical research: cell and gene therapy. [The Harvard Gazette] Press Release | |
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POLICY NEWSScience Funders Gamble on Grant Lotteries The Health Research Council of New Zealand is one of a growing number of funders that award grants partly through random selection. Earlier this year, for example, David Ackerley, a biologist at Victoria University of Wellington, received NZ$150,000 (US$96,000) to develop new ways to eliminate cells – after his number came up in the council’s annual lottery. [Nature News] Editorial Foreign Interference Fears Prompt Guidelines for Australian Universities New guidelines will help Australian universities protect themselves against foreign interference, says the country’s government. The advice follows concerns that foreign groups or governments, such as the government of China, might be seeking to instigate campus activities that are against Australia’s interests. [Nature News] Editorial
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EVENTSNEW Advancing Precision Medicine Drug Development: Incorporation of Real-World Data and Other Novel Strategies Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Junior Research Fellow – Synthetic Biology (Dresden University of Technology) Post-doctoral fellow – Cancer Immunology/Bioengineering (West Virginia University) Research Fellow/Research Associate – Cell & Gene Therapy (King’s College London) Research Specialist – Stem Cell Research (University of Illinois) Postdoctoral Fellow – Cancer Research (BC Cancer) Research Assistant – Cancer Research (BC Cancer) Postdoctoral Fellow – Cancer Research (Baylor College of Medicine) Postdoctoral Fellow – Cancer Therapy (Purdue University) Postdoctoral Fellow – Single Cell Genomics (BC Cancer) 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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