Functional rescue in an Angelman syndrome model following treatment with lentivector transduced hematopoietic stem cells
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Angelman Syndrome is a uncommon neurodevelopmental dysfunction characterised by impaired communication expertise, ataxia, motor and steadiness deficits, mental disabilities, and seizures. The genetic reason for Angelman syndrome is the neuronal lack of UBE3A expression within the mind. A novel method, described right here, is a stem cell gene remedy which makes use of lentivector transduced…
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An engineered novel lentivector specifically transducing dendritic cells and eliciting robust HBV-specific CTL response by upregulating autophagy in T cells.
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Dendritic cells (DCs) play a predominant function in initiating cell immune responses. Right here we generated a DC-targeting lentiviral vector (LVDC-UbHBcAg-LIGHT) and evaluated its capability to elicit HBV-specific cytotoxic T lymphocyte (CTL) responses. DC-SIGN-mediated particular transduction utilizing this assemble was confirmed in DC-SIGN-expressing 293T cells and ex vivo-cultured bone marrow cells. LVDC-UbHBcAg-LIGHT-loaded DCs have been…
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Lentivector Knockdown of CCR5 in Hematopoietic Stem and Progenitor Cells Confers Functional and Persistent HIV-1 Resistance in Humanized Mice.
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Gene-engineered CD34(+) hematopoietic stem and progenitor cells (HSPCs) can be utilized to generate an HIV-1-resistant immune system. Nevertheless, a sure threshold of transduced HSPCs is perhaps required for transplantation into mice for creating an HIV-resistant immune system. On this examine, we mixed CCR5 knockdown by a extremely environment friendly microRNA (miRNA) lentivector with pretransplantation number…
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Direct Lymph Node Vaccination of Lentivector/Prostate-Specific Antigen is Safe and Generates Tissue-Specific Responses in Rhesus Macaques.
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Anti-cancer immunotherapy is rising from a nadir and demonstrating tangible advantages to sufferers. A wide range of approaches are actually employed. We’re invoking antigen (Ag)-specific responses via direct injections of recombinant lentivectors (LVs) that encode sequences for tumor-associated antigens into a number of lymph nodes to optimize immune presentation/stimulation. Right here we first exhibit the…
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Concurrent mutations in RNA-dependent RNA polymerase and spike protein emerged as the epidemiologically most successful SARS-CoV-2 variant
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The D614G mutation within the Spike protein of the SARS-CoV-2 has successfully changed the early pandemic-causing variant. Utilizing pseudotyped lentivectors, we confirmed that the aspartate substitute by glycine in place 614 is markedly extra infectious. Molecular modelling means that the G614 mutation facilitates transition in the direction of an open state of the Spike protein. To…
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Highly efficient ‘hit-and-run’ genome editing with unconcentrated lentivectors carrying Vpr.Prot.Cas9 protein produced from RRE-containing transcripts
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The appliance of gene-editing know-how is presently restricted by the shortage of secure and environment friendly strategies to ship RNA-guided endonucleases to focus on cells. We engineered lentivirus-based nanoparticles to co-package the U6-sgRNA template and the CRISPR-associated protein 9 (Cas9) fused with a virion-targeted protein Vpr (Vpr.Prot.Cas9), for simultaneous supply to cells. Equal spatiotemporal management…
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