On the basis of our previous studies on the role and characterization of megakaryocytic MPs, and their ability to specifically target and deliver their cargo to HSPCs (27, 28), the aim of this study was to develop a system to deliver pDNA and small RNAs to HSPCs using human MkMPs. We examined functional delivery of pDNA using labeled pDNA and GFP protein expression. We also evaluated the delivery of two types of small RNA, siRNA and miRNA, using functional assays. All experiments were carried out with at least three biological and multiple technical replicates for statistical significance.

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