Category Archives: Cell Therapy

Cell Therapy News is an online publication and email newsletter dedicated to sharing the latest cell therapy, gene therapy, and regenerative medicine research.
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Published weekly since 2002, Cell Therapy News is our longest running online publication. Our editorial team curates the top publications, reviews, and news on in vivo gene therapy, cell therapy, and immunotherapy research. With over 15,000 subscribers and followers across our website, email newsletter, and Twitter feed, we are proud to facilitate the communication of cutting-edge cell therapy innovations.

Translation Elongation Factor 2 Depletion by siRNA in Mouse Liver Leads to mTOR-Independent Translational Upregulation of Ribosomal Protein Genes

Researchers demonstrated the feasibility of perturbing protein synthesis in a mouse liver by targeting translation elongation factor 2 with RNAi. They were able to achieve over 90% knockdown efficacy and maintain it for two weeks effectively slowing down the rate of translation elongation.
[Scientific Reports]
Gerashchenko, M. V., Nesterchuk, M. V., Smekalova, E. M., Paulo, J. A., Kowalski, P. S., Akulich, K. A., Bogorad, R., Dmitriev, S. E., Gygi, S., Zatsepin, T., Anderson, D. G., Gladyshev, V. N., & Koteliansky, V. E. (2020). Translation elongation factor 2 depletion by siRNA in mouse liver leads to mTOR-independent translational upregulation of ribosomal protein genes. Scientific Reports, 10(1), 15473. https://doi.org/10.1038/s41598-020-72399-4 Cite
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LRP6 Downregulation Promotes Cardiomyocyte Proliferation and Heart Regeneration

Gene therapy using the adeno-associated virus 9 miRNAi-Lrp6 construct promoted the repair of heart injury in mice.
[Cell Research]
Wu, Y., Zhou, L., Liu, H., Duan, R., Zhou, H., Zhang, F., He, X., Lu, D., Xiong, K., Xiong, M., Zhuang, J., Liu, Y., Li, L., Liang, D., & Chen, Y.-H. (2020). LRP6 downregulation promotes cardiomyocyte proliferation and heart regeneration. Cell Research, 1–13. https://doi.org/10.1038/s41422-020-00411-7 Cite
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CAR T Cell Therapies for Patients with Multiple Myeloma

Scientists highlight potential future innovations that could enhance the efficacy and/or reduce the toxicities associated with CAR T cell therapies.
[Nature Reviews Clinical Oncology]
CAR T cell therapies for patients with multiple myeloma | Nature Reviews Clinical Oncology. (n.d.). Retrieved September 25, 2020, from https://www.nature.com/articles/s41571-020-0427-6 Cite
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LRP6 Downregulation Promotes Cardiomyocyte Proliferation and Heart Regeneration

Researchers investigated the role of lipoprotein receptor-related protein 6 (LRP6) in heart repair through regulation of cardiomyocyte proliferation. Lrp6 deficiency increased cardiomyocyte cell cycle activity in neonatal, juvenile and adult mice.
[Cell Research]
Wu, Y., Zhou, L., Liu, H., Duan, R., Zhou, H., Zhang, F., He, X., Lu, D., Xiong, K., Xiong, M., Zhuang, J., Liu, Y., Li, L., Liang, D., & Chen, Y.-H. (2020). LRP6 downregulation promotes cardiomyocyte proliferation and heart regeneration. Cell Research, 1–13. https://doi.org/10.1038/s41422-020-00411-7 Cite
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Athenex Announces FDA Allowance of IND Application for TCRT-ESO-A2, a TCR-T Cell Therapy

Athenex, Inc. announced that the FDA has allowed its Investigational New Drug application for TCRT-ESO-A2, an autologous T cell receptor-T cell therapy targeting solid tumors that are NY-ESO-1 positive in HLA-A*02:01 positive patients.
[Athenex, Inc.]
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Seelos Therapeutics Announces Sponsored Research Agreement with Duke University for Gene Therapy Studies of SLS-004 in Parkinson’s Disease

Seelos Therapeutics, Inc. announced the signing of a Sponsored Research Agreement with Duke University to use the MPTP-induced Parkinson’s Disease (PD) mouse model to establish in vivo proof-of-concept study to demonstrate that administration of LV-dCas9-DNMT3A virus can prevent and/or delay PD and test the efficacy and safety of SLS-004. Subsequently, other pre-clinical models would be utilized to further validate the investigational product.
[Seelos Therapeutics, Inc.]
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Augmentation of NK Cell Proliferation and Anti-Tumor Immunity by Transgenic Expression of Receptors for Erythropoietin or Thrombopoietin

To reduce or preclude the necessity for IL-2 use, scientists investigated whether genetic engineering of NK cells to express the erythropoietin receptor or thrombopoietin receptor could be used as a method to improve NK cell survival and function.
[Molecular Therapy]
Chanswangphuwana, C., Allan, D. S. J., Chakraborty, M., Reger, R. N., & Childs, R. W. (2020). Augmentation of NK Cell Proliferation and Anti-tumor Immunity by Transgenic Expression of Receptors for Erythropoietin or Thrombopoietin. Molecular Therapy, 0(0). https://doi.org/10.1016/j.ymthe.2020.09.023 Cite
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Papyrus Therapeutics Inc. Signs Research Collaboration with Oxford Biomedica

Papyrus Therapeutics, Inc. and Oxford Biomedica plc announced the signing of a research collaboration agreement, which includes the assessment of the impact and therapeutic benefit of Papyrus’ PYTX-002, a potential first-in-class gene replacement therapy.
[Papyrus Therapeutics, Inc.]
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Tmunity Announces First Patient Dosed in Phase 1 Clinical Trial with CART-TnMUC1

Tmunity Therapeutics, Inc. announced that it has dosed the first patient in its Phase I CART-TnMUC1-01 clinical trial with the Tn/STn glycoform of mucin 1 chimeric antigen receptor T-cell therapy in patients with TnMUC1-positive advanced cancers.
[Tmunity Therapeutics, Inc.]
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Efficacy of Anti-CD147 Chimeric Antigen Receptors Targeting Hepatocellular Carcinoma

The authors report that T and NK cells transduced with a chimeric antigen receptor that recognize the surface marker, CD147, also known as Basigin, can effectively kill various malignant hepatocellular carcinoma (HCC) cell lines in vitro, and HCC tumors in xenograft and patient-derived xenograft mouse models.
[Nature Communications]
Tseng, H., Xiong, W., Badeti, S., Yang, Y., Ma, M., Liu, T., Ramos, C. A., Dotti, G., Fritzky, L., Jiang, J., Yi, Q., Guarrera, J., Zong, W.-X., Liu, C., & Liu, D. (2020). Efficacy of anti-CD147 chimeric antigen receptors targeting hepatocellular carcinoma. Nature Communications, 11(1), 4810. https://doi.org/10.1038/s41467-020-18444-2 Cite
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Stem/Progenitor Cell-Based Transplantation for Retinal Degeneration: A Review of Clinical Trials

Scientists provide a comprehensive overview on the progression of clinical trials for retinal degeneration treatment using four types of stem/progenitor cell-based transplantation to replace degenerative retinal cells and/or to supplement trophic factors from the aspects of safety, effectiveness and their respective advantages and disadvantages.
[Cell Death & Disease]
Wang, Y., Tang, Z., & Gu, P. (2020). Stem/progenitor cell-based transplantation for retinal degeneration: a review of clinical trials. Cell Death & Disease, 11(9), 1–14. https://doi.org/10.1038/s41419-020-02955-3 Cite
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