 | | Vol. 22.06 – 22 February, 2021 |
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| Researchers report a case of irreversible B cell maturation antigen (BCMA) loss in a patient with multiple myeloma who was enrolled in the KarMMa trial and progressed after anti-BCMA CAR T cell therapy.
[Nature Medicine] |
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 | | PUBLICATIONSRanked by the impact factor of the journal |
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| Scientists conducted a first-in-human, Phase I clinical trial of T cells engineered with a T cell receptor targeting HPV-16 E7 for the treatment of metastatic human papilloma virus-associated epithelial cancers.
[Nature Medicine] |
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| The authors combined induced pluripotent stem cell (iPSC) and CRISPR-Cas9 technologies to develop a model of the clonal evolution of acute myeloid leukemia.
[Cell Stem Cell] |
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| The engineered vectors elicited markedly reduced innate immune and T cell responses and enhanced gene expression in clinically relevant mouse and pig models across different tissues
[Science Translational Medicine] |
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| Scientists revealed previously unidentified insights into the modulation of macrophages to regulate tumor-associated fibrosis, providing a feasible strategy to reverse the immunosuppressive environment and improve the therapeutic outcome of checkpoint immunotherapies.
[Science Advances] |
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| The authors showed that expression of the glucagon-like peptide-1 gene delivered by epidermal stem cells attenuated development and reinstatement of alcohol-induced drug-taking and seeking as well as voluntary oral alcohol consumption.
[Molecular Psychiatry] |
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| Researchers demonstrated that a subset of tumor-associated macrophages that express folate receptor β possessed an immunosuppressive tumor progression profile.
[Nature Communications] |
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| The allogeneic Epstein-Barr virus (EBV)-specific T cells efficiently recognized human leukocyte antigen-matched EBV-encoded nuclear antigen 1-expressing and/or latent membrane proteins 1 and 2-expressing malignant cells and demonstrated therapeutic potential in a number of in vivo models.
[Journal for ImmunoTherapy of Cancer] |
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| Investigators explored systemic immune changes in 11 subjects with XLRS in a Phase I/IIa AAV8-RS1 gene therapy trial. Immune cell proportions and serum analytes were compared to 12 healthy male controls.
[Molecular Therapy] |
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| Researchers determined whether combined transplantation of human induced pluripotent stem cell-derived retinal precursor cells (RPC) and retinal pigment epithelial (RPE) cells was superior to RPE or RPC transplantation alone in preserving retinal from degeneration.
[Molecular Therapy-Methods & Clinical Development] |
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| Scientists review the principles by which the knowledge of genetics and genome engineering will drive the next generation of adoptive T cell therapies.
[Nature Reviews Genetics] |
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| The authors compare various in vitro cytotoxicity assays with respect to their experimental setup, appropriate uses, advantages, and disadvantages, and measures to overcome limitations. They also highlight the FDA directives for a potency assay for release of clinical cell therapy products.
[Nature Protocols] |
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| Investigators outline promising future directions for deciphering sensorineural hearing loss using disruptive molecular and genomic technologies.
[Stem Cells] |
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| Forge Biologics, Inc. announced that the FDA has granted Fast Track, Orphan Drug, and Rare Pediatric Disease (RPD) designations to FBX-101 for the treatment of patients with Krabbe disease. Forge is now actively recruiting patients for enrollment in the RESKUE phase I/II clinical trial of FBX-101, a novel, first-in-human AAV gene therapy for the disease.
[Forge Biologics, Inc.] |
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| March 2 – 3, 2021
Virtual |
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| | Swammerdam Institute for Life Sciences – Amsterdam, The Netherlands |
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| | Max Planck Institute of Molecular Physiology – Dortmund, Germany |
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| | University of California San Francisco – San Francisco, California, United States |
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| | NIH National Cancer Institute – Bethesda, Maryland, United States |
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| | The University of British Columbia – Vancouver, British Columbia, Canada |
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