T Cell-Intrinsic Role for Nod2 in Protection Against Th17-Mediated Uveiti

Scientists report a non-conventional, T cell-intrinsic function for Nod2 in suppression of Th17 immunity and experimental uveitis.
[Nature Communications]
Napier, R. J., Lee, E. J., Davey, M. P., Vance, E. E., Furtado, J. M., Snow, P. E., Samson, K. A., Lashley, S. J., Brown, B. R., Horai, R., Mattapallil, M. J., Xu, B., Callegan, M. C., Uebelhoer, L. S., Lancioni, C. L., Vehe, R. K., Binstadt, B. A., Smith, J. R., Caspi, R. R., & Rosenzweig, H. L. (2020). T cell-intrinsic role for Nod2 in protection against Th17-mediated uveitis. Nature Communications, 11(1), 5406. https://doi.org/10.1038/s41467-020-18961-0 Cite
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FoxO Maintains a Genuine Muscle Stem-Cell Quiescent State Until Geriatric Age

Interventions to neutralize Akt and promote FoxO activity drive a primed-to-genuine state conversion, whereas FoxO inactivation deteriorated the genuine state at a young age, causing regenerative failure of muscle, as occurs in geriatric mice.
[Nature Cell Biology]
García-Prat, L., Perdiguero, E., Alonso-Martín, S., Dell’Orso, S., Ravichandran, S., Brooks, S. R., Juan, A. H., Campanario, S., Jiang, K., Hong, X., Ortet, L., Ruiz-Bonilla, V., Flández, M., Moiseeva, V., Rebollo, E., Jardí, M., Sun, H.-W., Musarò, A., Sandri, M., … Muñoz-Cánoves, P. (2020). FoxO maintains a genuine muscle stem-cell quiescent state until geriatric age. Nature Cell Biology, 1–12. https://doi.org/10.1038/s41556-020-00593-7 Cite
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Targeted CRISPR Screening Identifies PRMT5 As Synthetic Lethality Combinatorial Target wth Gemcitabine in Pancreatic Cancer Cellsescent State Until Geriatric Age

Scientists performed an in vivo CRISPR screen in an orthotopic patient-derived xenograft model to identify gene targets whose inhibition creates synergistic tumor growth inhibition with gemcitabine.
[Proceedings of the National Academy of Sciences of the United States of America]
Wei, X., Yang, J., Adair, S. J., Ozturk, H., Kuscu, C., Lee, K. Y., Kane, W. J., O’Hara, P. E., Liu, D., Demirlenk, Y. M., Habieb, A. H., Yilmaz, E., Dutta, A., Bauer, T. W., & Adli, M. (2020). Targeted CRISPR screening identifies PRMT5 as synthetic lethality combinatorial target with gemcitabine in pancreatic cancer cells. Proceedings of the National Academy of Sciences. https://doi.org/10.1073/pnas.2009899117 Cite
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EdiGene Announces IND Application for Its CRISPR/Cas 9 Gene-Editing Hematopoietic Stem Cell Therapy ET-01 in β-Thalassemia Accepted for Review by China National Medical Products Administration

EdiGene, Inc. announced the Center for Drug Evaluation of China National Medical Products Administration has accepted for review the company’s Investigational New Drug application for ET-01, autologous CD34+ hematopoietic stem/progenitor cells with the erythroid -specific enhancer of the BCL11A gene modified by CRISPR/Cas9, an investigational gene-editing therapy for patients with transfusion dependent β-thalassemia.
[EdiGene, Inc.]
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Sumitomo Dainippon Pharma Oncology Announces First Patient Dosed in the Dubermatinib Arm of The Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial in Patients with Acute Myeloid Leukemia

Sumitomo Dainippon Pharma Oncology, Inc. announced the first patient has been dosed in a Phase Ib/II study evaluating the oral investigational agent, dubermatinib, an AXL kinase inhibitor, in combination with decitabine, in patients 60 years or older with newly diagnosed acute myeloid leukemia who have TP53 mutations and/or complex karyotype.
[Sumitomo Dainippon Pharma Oncology, Inc.]
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NexImmune Establishes Research Initiative with City of Hope to Focus on Novel Immunotherapeutic Approaches to Acute Myeloid Leukemia

NexImmune announced that it has signed a research initiative related to its AIM nanoparticle technology with City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases.
[NexImmune]
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Bayer Acquires Asklepios BioPharmaceutical to Broaden Innovation Base in Cell and Gene Therapy

Bayer AG announced the acquisition of Asklepios BioPharmaceutical, Inc., a US-headquartered biopharmaceutical company specialized in the research, development and manufacturing of gene therapies across different therapeutic areas.
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High-Throughput Continuous-Flow Microfluidic Electroporation of mRNA Into Primary Human T Cells for Applications in Cellular Therapy Manufacturing

Scientists present a microfluidic continuous-flow electrotransfection device designed for precise, consistent, and high-throughput genetic modification of target cells in cellular therapy manufacturing applications.
[Scientific Reports]
High-throughput continuous-flow microfluidic electroporation of mRNA into primary human T cells for applications in cellular therapy manufacturing | Scientific Reports. (n.d.). Retrieved October 22, 2020, from https://www.nature.com/articles/s41598-020-73755-0 Cite
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Quantitative PCR Methodology With a Volume-Based Unit for the Sophisticated Cellular Kinetic Evaluation of Chimeric Antigen Receptor T Cells

Researchers established a novel qPCR methodology incorporating a spike-in calibration curve that expressed cellular kinetics in units of copies/μL blood, as is the case for conventional pharmacokinetic studies of small molecules and other biologics.
[Scientific Reports]
Yamamoto, S., Matsumoto, S., Goto, A., Ugajin, M., Nakayama, M., Moriya, Y., & Hirabayashi, H. (2020). Quantitative PCR methodology with a volume-based unit for the sophisticated cellular kinetic evaluation of chimeric antigen receptor T cells. Scientific Reports, 10(1), 17884. https://doi.org/10.1038/s41598-020-74927-8 Cite
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Brainstem Development Requires Galactosylceramidase and Is Critical for Pathogenesis in a Model of Krabbe Disease

Using a novel Galc floxed allele, researchers induced ubiquitous galactosylceramidase ablation at various postnatal timepoints and identified a critical period of vulnerability to galactosylceramidase ablation between P4-6 in mice.
[Nature Communications]
Weinstock, N. I., Kreher, C., Favret, J., Nguyen, D., Bongarzone, E. R., Wrabetz, L., Laura Feltri, M., & Shin, D. (2020). Brainstem development requires galactosylceramidase and is critical for pathogenesis in a model of Krabbe disease. Nature Communications, 11(1), 5356. https://doi.org/10.1038/s41467-020-19179-w Cite
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Branched-Chain Amino Acid Aminotransferase 2 Regulates Ferroptotic Cell Death in Cancer Cells

By using a high-throughput CRISPR/Cas9-based genetic screen in HepG2 hepatocellular carcinoma cells to search for metabolic proteins inhibiting ferroptosis, researchers identified a branched-chain amino acid aminotransferase 2 as a novel suppressor of ferroptosis.
[Cell Death & Differentiation]
Wang, K., Zhang, Z., Tsai, H., Liu, Y., Gao, J., Wang, M., Song, L., Cao, X., Xu, Z., Chen, H., Gong, A., Wang, D., Cheng, F., & Zhu, H. (2020). Branched-chain amino acid aminotransferase 2 regulates ferroptotic cell death in cancer cells. Cell Death & Differentiation, 1–15. https://doi.org/10.1038/s41418-020-00644-4 Cite
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