Ligand-Induced Degradation of a CAR Permits Reversible Remote Control of CAR T Cell Activity In Vitro and In Vivo

The authors created a chimeric antigen-receptor (CAR) that was capable of on-demand downregulation by fusing the CAR to a previously developed ligand-induced degradation domain. Addition of a small molecule ligand triggered exposure of a cryptic degron within the ligand-induced degradation domain (LID), resulting in proteasomal degradation of the CAR-LID fusion protein and loss of CAR on the surface of T cells.
[Molecular Therapy]
Ligand-induced degradation of a CAR permits reversible remote control of CAR T cell activity in vitro and in vivo: Molecular Therapy. (n.d.). Retrieved June 16, 2020, from https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(20)30294-X Cite
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Huge Open-Access Journal Deal Inked by University of California and Springer Nature

The University of California (UC) system announced it has signed the biggest open-access deal in North America with one of the largest commercial scientific publishers. The agreement with Springer Nature includes a commitment by the publisher to explore making all articles that UC corresponding authors publish in the Nature family of journals immediately free to read on publication starting in 2022.
[ScienceInsider]
Editorial
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Quantitative Hybrid Cardiac [18F]FDG-PET-MRI Images for Assessment of Cardiac Repair by Pre-Conditioned Cardiosphere Derived Cells

Scientists enhanced the effect of cardiosphere-derived cells with APOSEC preconditioning (apoCDC), and investigated the reparative effect in a translational pig model of reperfused MI.
[Molecular Therapy-Methods & Clinical Development]
Winkler, J., Lukovic, D., Mester-Tonczar, J., Zlabinger, K., Gugerell, A., Pavo, N., Jakab, A., Szankai, Z., Traxler, D., Müller, C., Spannbauer, A., Riesenhuber, M., Hasimbegovic, E., Dawkins, J., Zimmermann, M., Ankersmit, H. J., Marbán, E., & Gyöngyösi, M. (2020). Quantitative hybrid cardiac [18F]FDG-PET-MRI images for assessment of cardiac repair by pre-conditioned cardiosphere derived cells. Molecular Therapy - Methods & Clinical Development, 0(0). https://doi.org/10.1016/j.omtm.2020.06.008 Cite
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Immunity-and-Matrix-Regulatory Cells Derived from Human Embryonic Stem Cells Safely and Effectively Treat Mouse Lung Injury and Fibrosis

Intravenous delivery of immunity- and matrix-regulatory cells, derived from human embryonic stem cells, inhibited both pulmonary inflammation and fibrosis in mouse models of lung injury, and significantly improved the survival rate of the recipient mice in a dose-dependent manner, likely through paracrine regulatory mechanisms.
[Cell Research]
Wu, J., Song, D., Li, Z., Guo, B., Xiao, Y., Liu, W., Liang, L., Feng, C., Gao, T., Chen, Y., Li, Y., Wang, Z., Wen, J., Yang, S., Liu, P., Wang, L., Wang, Y., Peng, L., Stacey, G. N., … Hao, J. (2020). Immunity-and-matrix-regulatory cells derived from human embryonic stem cells safely and effectively treat mouse lung injury and fibrosis. Cell Research, 1–16. https://doi.org/10.1038/s41422-020-0354-1 Cite
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UCLA Receives Nearly $14 Million from NIH to Investigate Gene Therapy to Combat HIV

UCLA researchers and colleagues have received a $13.65 million grant from the National Institutes of Health (NIH) to investigate and further develop an immunotherapy known as CAR T, which uses genetically modified stem cells to target and destroy HIV.
[University of California, Los Angeles]
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Hong Kong’s Contentious National Security Law Concerns Some Academics

China’s plan to introduce a new security law in Hong Kong has prompted mixed reactions from scientists there. Some fear that such a law could lead to government interference in research, restrict participation in international collaborations and increase self-censorship, whereas others are confident that research will be unaffected.
[Nature News]
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Long-Term Follow-Up of the First In Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 For Severe Hemophilia B

Scientists present longitudinal follow up data of subjects who participated in the first trial of a systemically-delivered adeno-associated viral (AAV) vector. Adult males with severe hemophilia B received an AAV2 vector at doses ranging from 8×1010 – 2×1012 vg/kg to target hepatocyte-specific expression of coagulation factor IX; a subset were followed for 12-15 years post vector administration.
[Molecular Therapy]
George, L. A., Ragni, M. V., Rasko, J. E. J., Raffini, L. J., Samelson-Jones, B., Hazbon, M., Runowski, A. R., Wellman, J. A., Wachtel, K., Chen, Y., Anguela, X. M., Kuranda, K., Mingozzi, F., & High, K. A. (2020). Long-Term Follow-Up of the First In Human Intravascular Delivery of AAV For Gene Transfer: AAV2-hFIX16 For Severe Hemophilia B. Molecular Therapy, 0(0). https://doi.org/10.1016/j.ymthe.2020.06.001 Cite
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Efficient CRISPR/Cas9-Mediated Gene Ablation in Human Keratinocytes to Recapitulate Genodermatoses: Modeling of Netherton Syndrome

Researchers have established a highly efficient method for precise deletion of critical gene sequences in primary human keratinocytes, based on CRISPR/Cas9-mediated gene editing.
[Molecular Therapy-Methods & Clinical Development]
Gálvez, V., Chacón-Solano, E., Bonafont, J., Mencía, Á., Di, W.-L., Murillas, R., Llames, S., Vicente, A., Rio, M. D., Carretero, M., & Larcher, F. (2020). Efficient CRISPR/Cas9-mediated gene ablation in human keratinocytes to recapitulate genodermatoses: Modeling of Netherton Syndrome. Molecular Therapy - Methods & Clinical Development, 0(0). https://doi.org/10.1016/j.omtm.2020.05.031 Cite
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Phase I Trial of Expanded, Activated Autologous NK Cell Infusions with Trastuzumab in Patients with HER2-Positive Cancers

The authors infused expanded, activated autologous natural killer cells to potentiate trastuzumab-mediated antibody-dependent cell cytotoxicity in patients with HER2-positive malignancies.
[Gene Therapy]
Lee, S.-C., Shimasaki, N., Lim, J. S. J., Wong, A. L., Yadav, K., Yong, W. P., Tan, L. K., Koh, L. P., Poon, M. L. M., Tan, S. H., Ow, S. G., Bharwani, L., Yap, Y.-S., Foo, M., Coustan-Smith, E., Sundar, R., Tan, H. L., Chong, W. Q., Kumarakulasinghe, N. B., … Campana, D. (2020). 1Phase I trial of expanded, activated autologous NK cell infusions with trastuzumab in patients with HER2-positive cancers. Clinical Cancer Research. https://doi.org/10.1158/1078-0432.CCR-20-0768 Cite
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Lentiviral Gene Therapy Rescues p47phox Chronic Granulomatous Disease and the Ability to Fight Salmonella Infection in Mice

Investigators tested the ability of a gene therapy approach to control infection by challenging p47phox-null mice with Salmonella Typhimurium, a leading cause of sepsis in chronic granulomatous diseasep atients, and found that mice reconstituted with lentivirus-transduced hematopoietic stem cells had a reduced bacterial load compared with untreated mice.
[Gene Therapy]
Schejtman, A., Aragão-Filho, W. C., Clare, S., Zinicola, M., Weisser, M., Burns, S. O., Booth, C., Gaspar, H. B., Thomas, D. C., Condino-Neto, A., Thrasher, A. J., & Santilli, G. (2020). Lentiviral gene therapy rescues p47 phox chronic granulomatous disease and the ability to fight Salmonella infection in mice. Gene Therapy, 1–11. https://doi.org/10.1038/s41434-020-0164-6 Cite
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Analysis of Aflibercept Expression in Non-Human Primates following Intravitreal Administration of ADVM-022, a Potential Gene Therapy for Neovascular Age-Related Macular Degeneration

Researchers explored whether ADVM-022, a recombinant adeno-associated virus vector designed to express aflibercept, could induce anti-VEGF protein levels comparable to those observed following a single bolus intravitreal injection of the standard-of-care aflibercept recombinant protein.
[Molecular Therapy-Methods & Clinical Development]
Kiss, S., Grishanin, R., Nguyen, A., Rosario, R., Greengard, J. S., Nieves, J., Gelfman, C. M., & Gasmi, M. (2020). Analysis of aflibercept expression in non-human primates following intravitreal administration of ADVM-022, a potential gene therapy for neovascular age-related macular degeneration. Molecular Therapy - Methods & Clinical Development, 0(0). https://doi.org/10.1016/j.omtm.2020.06.007 Cite
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