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Vectorology - GEG Tech top picks
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The authors used lentiviral vectors expressing the human perforin gene, under the transcriptional control of the ubiquitous PGK promoter or a lineage-specific perforin promoter, to correct the defect in different murine models. The results shown that mice with >30% engraftment of gene modified cells exhibited reduction of cytokine hypersecretion and cytopenias.
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