Emerging Role of Ubiquitination in the Regulation of PD-1/PD-L1 in Cancer Immunotherapy

Scientists reveal the development of a highly promising therapeutic approach for cancer immunotherapy by targeting PD-1/PD-L1 ubiquitination.
[Molecular Therapy]
Hu, X., Wang, J., Chu, M., Liu, Y., Wang, Z., & Zhu, X. (2020). Emerging role of ubiquitination in the regulation of PD-1/PD-L1 in cancer immunotherapy. Molecular Therapy, 0(0). https://doi.org/10.1016/j.ymthe.2020.12.032 Cite
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Layered Double Hydroxide as a Potent Non-Viral Vector for Nucleic Acid Delivery Using Gene-Activated Scaffolds for Tissue Regeneration Applications

Incorporation of layered double hydroxide–miRNA nanoparticles into collagen-nanohydroxyapatite scaffolds resulted in successful overexpression of miRNA in mesenchymal stromal cells, demonstrating the development of an efficacious miRNA delivery platform for gene therapy applications in regenerative medicine.
[Pharmaceutics]
Pharmaceutics | Free Full-Text | Layered Double Hydroxide as a Potent Non-viral Vector for Nucleic Acid Delivery Using Gene-Activated Scaffolds for Tissue Regeneration Applications | HTML. (n.d.). Retrieved January 4, 2021, from https://www.mdpi.com/1999-4923/12/12/1219/htm Cite
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Neonatal AAV Gene Therapy Rescues Hearing in a Mouse Model of SYNE4 Deafness

Researchers used AAV9‐PHP.B, a recently developed synthetic adeno‐associated virus, to deliver the coding sequence of Syne4 into the inner ears of neonatal Syne4−/− mice.
[EMBO Molecular Medicine]
Neonatal AAV gene therapy rescues hearing in a mouse model of SYNE4 deafness | EMBO Molecular Medicine. (n.d.). Retrieved January 4, 2021, from https://www.embopress.org/doi/full/10.15252/emmm.202013259 Cite
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Leukemic Extracellular Vesicles Induce Chimeric Antigen Receptor T Cell Dysfunction in Chronic Lymphocytic Leukemia

Scientists used chronic lymphocytic leukemia (CLL) as a model to investigate the interactions between the tumor microenvironment and CART cells.
[Molecular Therapy]
Cox, M. J., Lucien, F., Sakemura, R., Boysen, J. C., Kim, Y., Horvei, P., Roman, C. M., Hansen, M. J., Tapper, E. E., Siegler, E. L., Forsman, C., Crotts, S. B., Schick, K. J., Hefazi, M., Ruff, M. W., Can, I., Adada, M., Bezerra, E., Fonkoua, L. A. K., … Kenderian, S. S. (2020). Leukemic Extracellular Vesicles Induce Chimeric Antigen Receptor T Cell Dysfunction in Chronic Lymphocytic Leukemia. Molecular Therapy, 0(0). https://doi.org/10.1016/j.ymthe.2020.12.033 Cite
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F-star Therapeutics Announces First Patient Dosed in FS222 Phase I Clinical Trial

F-star Therapeutics, Inc. announced that the first patient has been dosed in its Phase I trial evaluating FS222, a potentially best-in-class bispecific antibody targeting CD137 and PD-L1.
[F-star Therapeutics, Inc. (Globe Newswire, Inc.)]
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Polyphenols and Neurodegenerative Diseases: Focus on Neuronal Regeneration

Scientists focus on the concepts of regeneration, hurdles faced in the path of regenerative therapy, neurodegenerative diseases and the idea of using peptides, cytokines, tissue engineering, genetic engineering, advanced stem cell therapy, and polyphenolic phytochemicals to cure damaged tissues and neurodegenerative diseases.
[Critical Reviews in Food Science and Nutrition]
Moradi, S. Z., Jalili, F., Farhadian, N., Joshi, T., Wang, M., Zou, L., Cao, H., Farzaei, M. H., & Xiao, J. (2021). Polyphenols and neurodegenerative diseases: focus on neuronal regeneration. Critical Reviews in Food Science and Nutrition, 0(0), 1–16. https://doi.org/10.1080/10408398.2020.1865870 Cite
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Lentiviral Delivery of Co-Packaged Cas9 mRNA and a Vegfa-Targeting Guide RNA Prevents Wet Age-Related Macular Degeneration in Mice

Scientists showed that the lentiviral codelivery of Streptococcus pyogenes Cas9 mRNA and expression cassettes that encoded a guide RNA that targeted vascular endothelial growth factor A (Vegfa) was efficacious in a mouse model of wet age-related macular degeneration induced by Vegfa.
[Nature Biomedical Engineering]
Ling, S., Yang, S., Hu, X., Yin, D., Dai, Y., Qian, X., Wang, D., Pan, X., Hong, J., Sun, X., Yang, H., Paludan, S. R., & Cai, Y. (2021). Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa -targeting guide RNA prevents wet age-related macular degeneration in mice. Nature Biomedical Engineering, 1–13. https://doi.org/10.1038/s41551-020-00656-y Cite
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Combined Lentiviral- and RNA-Mediated CRISPR/Cas9 Delivery for Efficient and Traceable Gene Editing in Human Hematopoietic Stem and Progenitor Cells

Researchers developed a combinatorial system using stable lentiviral delivery of single guide RNA followed by transient transfection of Cas9 mRNA by electroporation in human cord blood-derived CD34+ hematopoietic stem and progenitor cells.
[Scientific Reports]
Yudovich, D., Bäckström, A., Schmiderer, L., Žemaitis, K., Subramaniam, A., & Larsson, J. (2020). Combined lentiviral- and RNA-mediated CRISPR/Cas9 delivery for efficient and traceable gene editing in human hematopoietic stem and progenitor cells. Scientific Reports, 10(1), 22393. https://doi.org/10.1038/s41598-020-79724-x Cite
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Targeting USP47 Overcomes Tyrosine Kinase Inhibitor Resistance and Eradicates Leukemia Stem/Progenitor Cells in Chronic Myelogenous Leukemia

The knockout of Usp47 significantly inhibited BCR-ABL and BCR-ABLT315I-induced CML in mice with the reduction of Lin−Sca1+c-Kit+ chronic myelogenous leukemia stem/progenitor cells.
[Nature Communications]
Lei, H., Xu, H.-Z., Shan, H.-Z., Liu, M., Lu, Y., Fang, Z.-X., Jin, J., Jing, B., Xiao, X.-H., Gao, S.-M., Gao, F.-H., Xia, L., Yang, L., Liu, L.-G., Wang, W.-W., Liu, C.-X., Tong, Y., Wu, Y.-Z., Zheng, J.-K., … Wu, Y.-L. (2021). Targeting USP47 overcomes tyrosine kinase inhibitor resistance and eradicates leukemia stem/progenitor cells in chronic myelogenous leukemia. Nature Communications, 12(1), 51. https://doi.org/10.1038/s41467-020-20259-0 Cite
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Suppressing Sart1 To Modulate Macrophage Polarization by siRNA-Loaded Liposomes: A Promising Therapeutic Strategy for Pulmonary Fibrosis

Researchers report a potential nanomedicine-based gene therapy for idiopathic pulmonary fibrosis based on regulate macrophage polarization.
[Theranostics]
Pan, T., Zhou, Q., Miao, K., Zhang, L., Wu, G., Yu, J., Xu, Y., Xiong, W., Li, Y., & Wang, Y. (2021). Suppressing Sart1 to modulate macrophage polarization by siRNA-loaded liposomes: a promising therapeutic strategy for pulmonary fibrosis. Theranostics, 11(3), 1192–1206. https://doi.org/10.7150/thno.48152 Cite
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Human Umbilical Cord-Derived Mesenchymal Stem Cell Therapy Ameliorates Lupus through Increasing CD4+ T Cell Senescence via MiR-199a-5p/Sirt1/p53 Axis

MRL/lpr mice were injected with miR-199a-5p agomir to evaluate the effects of miR-199a-5p on splenic CD4+ T cell senescence and disease in vivo.
[Theranostics]
Cheng, T., Ding, S., Liu, S., Li, Y., & Sun, L. (2021). Human umbilical cord-derived mesenchymal stem cell therapy ameliorate lupus through increasing CD4+ T cell senescence via MiR-199a-5p/Sirt1/p53 axis. Theranostics, 11(2), 893–905. https://doi.org/10.7150/thno.48080 Cite
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