| Vol. 21.34 – 14 December, 2020 |
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| The role of cardiac immunoglobulin E (IgE) receptor (FcεR1) signaling in pathological cardiac remodeling was explored in vivo by FcεR1 genetic depletion, anti-IgE antibodies, and bone-marrow transplantation. The roles of IgE-FcεR1 pathway were further evaluated in vitro in primary cultured rat cardiomyocytes and cardiac fibroblasts. [Circulation] |
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| PUBLICATIONSRanked by the impact factor of the journal |
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| REVERSE was a randomized, double-masked, sham-controlled, multicenter, Phase III clinical trial that evaluated the efficacy of a single intravitreal injection of rAAV2/2-ND4 in subjects with visual loss from Leber hereditary optic neuropathy. [Science Translational Medicine] |
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| Using patient-derived cellular models, researchers showed that loss of ZFAND3 hampered the invasive capacity of glioblastoma, whereas ZFAND3 overexpression increased motility in cells that were initially not invasive. [Nature Communications] |
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| The authors demonstrated that Cas9-ribonucleoprotein-based genome editors could correct two distinct mutant alleles within a single human cell precisely. Gene-corrected cells in an iPSC model of Pompe disease expressed the corrected transcript from both corrected alleles, leading to enzymatic cross-correction of diseased cells. [Nature Communications] |
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| Researchers describe a simple method to obtain therapeutics-containing “dead cells” by shocking live cancer cells in liquid nitrogen to eliminate pathogenicity while preserving their major structure and chemotaxis toward the lesion site. [Science Advances] |
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| 49 patients received tyrosine kinase inhibitors monotherapy, while 91 received additional treatment. Toxicity of second-/third-generation tyrosine kinase inhibitors post allogeneic stem cell transplantation was comparable to pretransplant use and could be managed with dose reduction or temporary discontinuation. [Bone Marrow Transplantation] |
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| Scientists evaluated standard-of-care treatment with or without midostaurin to prevent relapse following allogeneic hematopoietic stem cell transplant in patients with acute myeloid leukemia harboring internal tandem duplication in FLT3. [Bone Marrow Transplantation] |
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| Investigators performed a multicenter retrospective cohort study of patients with T-cell lymphoblastic lymphoma treated using leukemia-type initial therapies to compare the outcomes after hematopoietic stem cell transplantation at different disease stages. [Scientific Reports] |
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| Researchers assessed the effects that menstrual blood-derived stromal cells had on the recruitment of macrophages and other innate immune cells in two mouse models of acute inflammation, a thioglycollate-elicited peritonitis model and a monobacterial sepsis model. [Scientific Reports] |
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| The authors review the most recent advances in hematopoietic stem and progenitor cell (HSPC) gene therapy and discuss emerging strategies for using HSPC gene therapy for a range of diseases. [Nature Reviews Genetics] |
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| It is a double milestone: new evidence that cures are possible for many people born with sickle cell disease and another serious blood disorder, beta-thalassemia, and a first for the genome editor CRISPR. [ScienceInsider] |
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| Precision BioSciences, Inc. announced positive interim clinical results from its Phase I/IIa study of PBCAR0191, the company’s off-the-shelf allogeneic CAR T cell therapy investigational candidate targeting CD19. 27 patients including 16 patients with aggressive NHL and 11 patients with aggressive B-ALL were enrolled and evaluated. [Precision BioSciences, Inc.] |
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| Cytovia Therapeutics announced that it plans to file with the FDA in 2021 an Investigational New Drug application and initiate clinical trials in hematological and solid tumors with its Universal iPSC NK cell therapy. [Cytovia Therapeutics (GlobeNewswire, Inc.)] |
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| January 25 – 27, 2021 Virtual |
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| Karolinska Institutet – Stockholm, Sweden |
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| Columbia University – New York, New York, United States |
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| H. Lee Moffitt Cancer Center & Research Institute – Tampa, Florida, United States |
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| Axovia Therapeutics – London, England, United Kingdom |
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| EnaraBio – Oxford, England, United Kingdom |
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