Gene therapy is the modification of gene expression to treat a disease. However, efficient intracellular delivery and monitoring of gene therapeutic agents is an ongoing challenge. Use of theranostic agents with suitable targeted, controlled delivery and imaging modalities has the potential to greatly advance gene therapy. Inorganic nanoparticles including magnetic nanoparticles, gold nanoparticles, and quantum dots have been shown to be effective theranostic agents for the delivery and spatiotemporal tracking of oligonucleotides in vitro and even a few cases in vivo. Major concerns remain to be addressed including cytotoxicity, particularly of quantum dots; effective dosage of nanoparticles for optimal theranostic effect; development of real-time in vivo imaging; and further improvement of gene therapy efficacy.

中文翻译：

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通过时空成像进行细胞内基因传递的治疗诊断剂

基因治疗是通过改变基因表达来治疗疾病。然而，基因治疗剂的有效细胞内递送和监测是一个持续的挑战。使用具有合适靶向、受控递送和成像方式的治疗诊断剂有可能极大地推进基因治疗。包括磁性纳米颗粒、金纳米颗粒和量子点在内的无机纳米颗粒已被证明是有效的治疗诊断剂，可用于体外甚至体内少数情况下寡核苷酸的递送和时空追踪。主要问题仍有待解决，包括细胞毒性，特别是量子点的细胞毒性；纳米粒子的有效剂量以获得最佳的治疗诊断效果；实时体内成像的发展；进一步提高基因治疗的疗效。