Non-invasive gene monitoring is important for most gene therapy applications to ensure selective gene transfer to specific cells or tissues. We developed a non-invasive imaging system to assess the location and persistence of gene expression by anchoring an anti-dansyl (DNS) single-chain antibody (DNS receptor) on the cell surface to trap DNS-derivatized imaging probes. DNS hapten was covalently attached to cross-linked iron oxide (CLIO) to form a 39+/-0.5 nm DNS-CLIO nanoparticle imaging probe. DNS-CLIO specifically bound to DNS receptors but not to a control single-chain antibody receptor. DNS-CLIO (100 microM Fe) was non-toxic to both B16/DNS (DNS receptor positive) and B16/phOx (control receptor positive) cells. Magnetic resonance (MR) imaging could detect as few as 10% B16/DNS cells in a mixture in vitro. Importantly, DNS-CLIO specifically bound to a B16/DNS tumor, which markedly reduced signal intensity. Similar results were also shown with DNS quantum dots, which specifically targeted CT26/DNS cells but not control CT26/phOx cells both in vitro and in vivo. These results demonstrate that DNS nanoparticles can systemically monitor the expression of DNS receptor in vivo by feasible imaging systems. This targeting strategy may provide a valuable tool to estimate the efficacy and specificity of different gene delivery systems and optimize gene therapy protocols in the clinic.

译文

非侵入性基因监测对于大多数基因治疗应用是重要的,以确保选择性基因转移到特定的细胞或组织。我们开发了一种非侵入性成像系统,通过将抗dansyl (DNS) 单链抗体 (DNS受体) 锚定在细胞表面以捕获DNS衍生的成像探针来评估基因表达的位置和持久性。DNS半抗原与交联的氧化铁 (CLIO) 共价连接以形成39 +/-0.5纳米DNS-CLIO纳米颗粒成像探针。DNS-CLIO与DNS受体特异性结合,但不与对照单链抗体受体结合。DNS-CLIO (100微微Fe) 对B16/DNS (DNS受体阳性) 和B16/phOx (对照受体阳性) 细胞均无毒。磁共振 (MR) 成像可以在体外检测到混合物中的10% 个B16/DNS细胞。重要的是,DNS-CLIO与B16/DNS肿瘤特异性结合,从而显着降低了信号强度。DNS量子点也显示了类似的结果,该量子点专门针对CT26/DNS细胞,但在体外和体内均不控制CT26/phOx细胞。这些结果表明,DNS纳米颗粒可以通过可行的成像系统系统地监测体内DNS受体的表达。这种靶向策略可能为评估不同基因递送系统的功效和特异性以及优化临床中的基因治疗方案提供有价值的工具。

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