 | | Vol. 22.07 – 1 March, 2021 |
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| Scientists developed an in vivo T cell CRISPR screening platform and identified a key mechanism restraining effector T cell biology through the ETS family TF, Fli1.
[Cell] |
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 | | PUBLICATIONSRanked by the impact factor of the journal |
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| The authors showed the generation of a CD105-specific nanobody, an anti-human CD105 CAR-T cells, by inserting the sequences for anti-CD105 nanobody-linked standard cassette genes into AAVS1 site using CRISPR/Cas9 technology.
[Signal Transduction and Targeted Therapy] |
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| Using T cell bispecific antibodies and chimeric antigen receptors directed against HER2, scientists showed that disruption of interferon-gamma signaling confers resistance to killing by active T lymphocytes.
[Nature Communications] |
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| Researchers developed CRISPR-edited universal off-the-shelf CD19/CD22 dual-targeted CAR-T cells as a novel therapy for relapsed/refractory acute lymphoblastic leukemia.
[Clinical Cancer Research] |
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| Integrative analysis identified 117 p30-dependent regulatory elements associated with 33 strongly down-regulated genes upon p30-knockdown. CRISPR/Cas9-mediated mutational disruption of these genes revealed the RNA-binding protein MSI2 as a critical p30-target.
[Leukemia] |
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| Investigators evaluated CAR-T cells specific for Siglec-6, an antigen expressed in chronic lymphocytic leukemia (CLL), as a novel CAR-T cell treatment for CLL.
[Leukemia] |
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| Scientists report the development of novel engineered recombinant adeno‐associated virus (rAAV) vectors that enabled efficient targeting of photoreceptors via less invasive intravitreal administration.
[EMBO Molecular Medicine] |
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| The authors used adeno-associated virus to deliver CRISPR/guide RNA designed to abolish the genes NeuN, GFAP, or MOG expressed specifically in neurons, astrocytes, or oligodendrocytes respectively in the central nervous system of mice.
[Gene Therapy] |
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| Researchers established a procedure for placental HSC transplantation into E11.5 Runx1-deficient mice mated with G1-HRD-Runx1 transgenic mice that had no HSCs in the fetal liver.
[Scientific Reports] |
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| Scientists investigated the influence of the UNC93B1 single-nucleotide polymorphism rs308328 on transplant outcomes in a cohort of 237 patients undergoing unrelated HLA-matched bone marrow transplantation.
[Genes & Immunity] |
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| Scientists describe the current clinical evidence of anti-CAR immune responses and discuss how new CAR T cell technologies might impact the risk of immunogenicity.
[Nature Reviews Clinical Oncology] |
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| Evaluation of research and clinical trials in gene therapy reveals a concentration of activity in prostate cancer research and practice.
[Nature Reviews Urology] |
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| The authors summarize the Layer-by-Layer approaches that can be effectively used for the gene delivery, considering various examples with the transfer of pDNA, mRNA, siRNA as well as genome-editing tools.
[Expert Opinion on Drug Delivery] |
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| AVROBIO, Inc. announced that the European Commission has granted orphan drug designation for AVR-RD-04, the company’s investigational gene therapy for the treatment of cystinosis.
[AVROBIO, Inc.] |
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| MaxiVAX SA and Minaris Regenerative Medicine GmbH have entered into a manufacturing agreement for MVX-ONCO-2, a cell-based immunotherapy for the treatment of cancers such as chordoma, head and neck, and other solid tumors.
[MaxiVAX SA] |
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| | Children’s Hospital of Philadelphia – Philadelphia, Pennsylvania, United States |
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| | Swammerdam Institute for Life Sciences – Amsterdam, The Netherlands |
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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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