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| Investigators demonstrated prime editing with purified ribonucleoprotein complexes. They introduced somatic mutations in zebrafish embryos with frequencies as high as 30% and demonstrated germline transmission.
[Nature Biotechnology] |
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 | | PUBLICATIONSRanked by the impact factor of the journal |
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| In immunodeficient mice bearing intracerebral patient-derived xenografts with heterogeneous expression of EGFRvIII, a single intravenous infusion of epidermal growth factor receptor splice variant III synNotch-CAR T cells demonstrated higher antitumor efficacy and T cell durability.
[Science Translational Medicine] |
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| Researchers improved the specificity and persistent antitumor activity of therapeutic T cells with synthetic Notch CAR circuits.
[Science Translational Medicine] |
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| Scientists identified major m6A “writers” as the top candidate genes regulating macrophage activation by LPS in an RNA binding protein focused CRISPR screening.
[Science Advances] |
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| The authors sought to engineer antigen-specific T regulatory cells to suppress antibody formation against the soluble therapeutic protein factor VIII in an MHC-independent fashion.
[Molecular Therapy] |
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| To minimize GM-CSF production from CART to reduce its associated toxicity, scientists conducted a pilot study of CRISPR-edited GM-CSF knockout (KO) in CART secreting anti-IL6 scFv and IL1RA, with additional TCR KO for tracing edited CART.
[Cell Discovery] |
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| Researchers evaluated the effect of allogenic mesenchymal stem cell transplantation in a large animal model of repeated biliary obstruction followed by partial hepatectomy.
[International Journal of Molecular Sciences] |
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| Scientists assessed the safety/efficacy of a tissue–engineered biocomplex in periodontal reconstruction.
[Journal of Periodontology] |
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| Scientists induced human umbilical cord-mesenchymal stem cells (hUC-MSCs) to differentiate into type 2 alveolar epithelial cells (AEC2s), and provided evidence that intratracheal transplantation of hUC-MSC-derived AEC2s could improve mortality and alleviate fibrosis in bleomycin-induced pulmonary fibrosis mice.
[Stem Cells and Development] |
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| The authors performed intravitreal injections of self-complementary AAV9 vectors packaged with either ovine CLN5 or CLN6 into one eye of three-month-old CLN5−/− or CLN6−/− animals, respectively.
[Experimental Eye Research] |
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| Investigators analyzed the outcome of this treatment osteonecrosis, the number and characteristics of bone marrow mesenchymal cells that could be transplanted, and the risks of tumorigenesis in these patients with hematologic cancers.
[International Orthopaedics] |
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| The authors summarize the existing engineered tumor models with a focus on tumor immunology and cell-based immunotherapy. They also discuss some key considerations for the future development of engineered tumor models for immunotherapeutics.
[Acta Biomaterialia] |
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| The basis for development, mechanism of action and outcomes from recently published Cas9-based clinical trial (genetic editing) and dCas9/Cas13-based pre-clinical (epigenetic editing) data are discussed.
[Molecular Therapy-Methods & Clinical Development] |
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| | Six patients with dementia traveled to Mexico last year to be injected with a gene therapy not authorized for use in the US, according to the CEO of a Seattle-area startup that wants to accelerate testing of unproven anti-aging medicines and views US drug safety regulations as a hindrance.
[STAT News] |
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Tessa Therapeutics Ltd. announced enrollment of 12 patient Pilot cohort of its Phase II trial in relapsed/refractory Classical Hodgkin Lymphoma (R/R cHL). The next step in the two-part study is enrollment of the 82 patient Pivotal cohort to assess the safety and antitumor efficacy of Tessa’s autologous CD30 CAR-T in R/R cHL.
[Tessa Therapeutics Ltd.] |
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| | St. Jude Children’s Research Hospital – Memphis, Tennessee, United States |
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| | Stanford University – Stanford, California, United States |
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| | Zhejiang University – Zhejiang, China |
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| | LSU Health Science Center – Shreveport, Louisianam United States |
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| | Rutgers University – New Brunswick, New Jersey, United States |
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