Pfizer Doses First Participant in Phase III Study for Duchenne Muscular Dystrophy Investigational Gene Therapy

Pfizer Inc. announced that the first participant has been dosed in the Phase III CIFFREO study, which will evaluate the efficacy and safety of investigational gene therapy candidate PF-06939926 in boys with Duchenne muscular dystrophy.
[Pfizer Inc.]
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CXCR5 CAR-T Cells Simultaneously Target B Cell Non-Hodgkin’s Lymphoma and Tumor-Supportive Follicular T Helper Cells

To target both B cell Non-Hodgkin’s lymphoma and follicular T helper cells in the tumor microenvironment, researchers applied a chimeric antigen receptor that recognized human CXCR5 with high avidity.
[Nature Communications]
Bunse, M., Pfeilschifter, J., Bluhm, J., Zschummel, M., Joedicke, J. J., Wirges, A., Stark, H., Kretschmer, V., Chmielewski, M., Uckert, W., Abken, H., Westermann, J., Rehm, A., & Höpken, U. E. (2021). CXCR5 CAR-T cells simultaneously target B cell non-Hodgkin’s lymphoma and tumor-supportive follicular T helper cells. Nature Communications, 12(1), 240. https://doi.org/10.1038/s41467-020-20488-3 Cite
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CRISPR Technologies for Precise Epigenome Editing

The authors review the state of currently achievable epigenetic manipulations along with corresponding applications. With future optimization, CRISPR-based epigenomic editing stands as a set of powerful tools for understanding and controlling biological function.
[Nature Cell Biology]
Nakamura, M., Gao, Y., Dominguez, A. A., & Qi, L. S. (2021). CRISPR technologies for precise epigenome editing. Nature Cell Biology, 23(1), 11–22. https://doi.org/10.1038/s41556-020-00620-7 Cite
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Dynamic Immune Profiling Identifies the Stronger Graft-Versus-Leukemia (GVL) Effects With Haploidentical Allografts Compared to HLA-Matched Stem Cell Transplantation

Researchers employed two nonirradiated leukemia mouse models which carried human AML-ETO or MLL-AF9 fusion gene to establish haplo-identical and major histocompatibility-matched transplantation models and investigated the immune cell dynamic response during leukemia development in vivo.
[Cellular & Molecular Immunology]
Guo, H., Chang, Y.-J., Hong, Y., Xu, L.-P., Wang, Y., Zhang, X.-H., Wang, M., Chen, H., Chen, Y.-H., Wang, F.-R., Wei-Han, Sun, Y.-Q., Yan, C.-H., Tang, F.-F., Mo, X.-D., Liu, K.-Y., & Huang, X.-J. (2021). Dynamic immune profiling identifies the stronger graft-versus-leukemia (GVL) effects with haploidentical allografts compared to HLA-matched stem cell transplantation. Cellular & Molecular Immunology, 1–14. https://doi.org/10.1038/s41423-020-00597-1 Cite
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CRISPR/Cas9 Directed to the Ube3a Antisense Transcript Improves Angelman Syndrome Phenotype in Mice

Scientists showed that gene editing of Ube3a-ATS in the mouse brain resulted in the formation of base pair insertions/deletions in neurons and the subsequent unsilencing of the paternal Ube3a allele in neurons, which partially corrected the behavior phenotype of a murine Angelman syndrome model.
[Journal of Clinical Investigation]
Schmid, R. S., Deng, X., Panikker, P., Msackyi, M., Breton, C., & Wilson, J. M. (2021). CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice. The Journal of Clinical Investigation. https://doi.org/10.1172/JCI142574 Cite
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A Modular and Controllable T Cell Therapy Platform for Acute Myeloid Leukemia

Researchers developed a modular and controllable MHC-unrestricted adoptive T cell therapy platform tailored to acute myeloid leukemia.
[Leukemia]
Benmebarek, M.-R., Cadilha, B. L., Herrmann, M., Lesch, S., Schmitt, S., Stoiber, S., Darwich, A., Augsberger, C., Brauchle, B., Rohrbacher, L., Oner, A., Seifert, M., Schwerdtfeger, M., Gottschlich, A., Rataj, F., Fenn, N. C., Klein, C., Subklewe, M., Endres, S., … Kobold, S. (2021). A modular and controllable T cell therapy platform for acute myeloid leukemia. Leukemia, 1–15. https://doi.org/10.1038/s41375-020-01109-w Cite
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U2af1 Is Required for Survival and Function of Hematopoietic Stem/Progenitor Cells

Using a novel conditional U2af1 knockout allele, investigators found that deletion of U2af1 results in profound defects in hematopoiesis characterized by pancytopenia, ablation of hematopoietic stem/progenitor cells leading to bone marrow failure and early lethality in mice.
[Leukemia]
Dutta, A., Yang, Y., Le, B. T., Zhang, Y., Abdel-Wahab, O., Zang, C., & Mohi, G. (2021). U2af1 is required for survival and function of hematopoietic stem/progenitor cells. Leukemia, 1–17. https://doi.org/10.1038/s41375-020-01116-x Cite
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Overcoming Target Epitope Masking Resistance that Can Occur on Low-Antigen-Expresser AML Blasts after IL-1RAP Chimeric Antigen Receptor T Cell Therapy Using the Inducible Caspase 9 Suicide Gene Safety Switch

As acute myeloid leukemia (AML) primary blasts express different levels of accessory protein of the interleukin-1 receptor (IL-1RAP), scientists modeled transduction of different AML tumor cell lines screened for density of antigenic sites with their lentiviral vectors carrying a third-generation IL-1RAP chimeric antigen receptor, an iCASP9 suicide gene, and a truncated CD19 surface gene.
[Cancer Gene Therapy]
Warda, W., Da Rocha, M. N., Trad, R., Haderbache, R., Salma, Y., Bouquet, L., Roussel, X., Nicod, C., Deschamps, M., & Ferrand, C. (2021). Overcoming target epitope masking resistance that can occur on low-antigen-expresser AML blasts after IL-1RAP chimeric antigen receptor T cell therapy using the inducible caspase 9 suicide gene safety switch. Cancer Gene Therapy, 1–11. https://doi.org/10.1038/s41417-020-00284-3 Cite
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HUCMSCs Transplantation Combined with Ultrashort Wave Therapy Attenuates Neuroinflammation in Spinal Cord Injury through NUR77/ NF-κB Pathway

Low-dose Ultrashort Wave was treated one day after spinal cord injury (SCI) and human umbilical cord mesenchymal stem cell suspension was transferred to the lesion using a micro-syringe seven days after SCI.
[Life Sciences]
Wang, S., Jia, Y., Cao, X., Feng, S., Na, L., Dong, H., Gao, J., & Zhang, L. (2020). HUCMSCs transplantation combined with ultrashort wave therapy attenuates neuroinflammation in spinal cord injury through NUR77/ NF-κB pathway. Life Sciences, 118958. https://doi.org/10.1016/j.lfs.2020.118958 Cite
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Radiation Impacts Early Atherosclerosis by Suppressing Intimal LDL Accumulation

Atherosclerosis was studied in the ascending aortic arch and compared to mice without bone marrow transplantation (BMT). In BMT mice neutral lipid and myeloid cell topography were lower in lesions after feeding a cholesterol-rich diet for three, six and 12 weeks.
[Circulation Research]
Ikeda Jiro, Scipione Corey A, Hyduk Sharon, Althagafi Marwan G, Atif Jawairia, Dick Sarah A, Rajora Maneesha A, Jang Erika, Emoto Takuo, Murakami Junichi, Ikeda Noriko, Ibrahim Hisham M, Polenz Chanele K, Gao Xiaotang, Tai Kelly, Jongstra-Bilen Jenny, Nakashima Ryota, Epelman Slava, Robbins Clinton, … Cybulsky Myron I. (n.d.). Radiation Impacts Early Atherosclerosis by Suppressing Intimal LDL Accumulation. Circulation Research, 0(0). https://doi.org/10.1161/CIRCRESAHA.119.316539 Cite
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Platelet-Targeted Gene Therapy Induces Robust Immune Tolerance Even in a Primed Model via Peripheral Clonal Deletion of Antigen-Specific CD4 and CD8 T Cells and Expansion of Antigen-Specific Regulatory T Cells

Using the OT-I/WT model, we showed that antigen-specific CD8 T cells were partially deleted in recipients after platelet-targeted gene transfer.
[Molecular Therapy-Nucleic Acids]
Li, J., Chen, J., Schroeder, J. A., Hu, J., Williams, C. B., & Shi, Q. (2021). Platelet-targeted gene therapy induces robust immune tolerance even in a primed model via peripheral clonal deletion of antigen-specific CD4 and CD8 T cells and expansion of antigen-specific regulatory T cells. Molecular Therapy - Nucleic Acids, 0(0). https://doi.org/10.1016/j.omtn.2020.12.026 Cite
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