Knipe Jennifer M, Peters Jonathan T, Peppas Nicholas A
Department of Chemical Engineering, C0400, The University of Texas at Austin, Austin, TX 78712 (USA).
Nano Today. 2013 Feb 1;8(1):21-38. doi: 10.1016/j.nantod.2012.12.004.
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 and even a few cases . Major concerns remain to be addressed including cytotoxicity, particularly of quantum dots; effective dosage of nanoparticles for optimal theranostic effect; development of real-time imaging; and further improvement of gene therapy efficacy.
基因治疗是通过改变基因表达来治疗疾病。然而,基因治疗药物的高效细胞内递送和监测仍是一个持续存在的挑战。使用具有合适靶向、可控递送和成像方式的诊疗试剂有可能极大地推动基因治疗的发展。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用于寡核苷酸递送和时空追踪的有效诊疗试剂,甚至在少数情况下也是如此。仍有一些主要问题有待解决,包括细胞毒性,尤其是量子点的细胞毒性;纳米颗粒的有效剂量以实现最佳诊疗效果;实时成像的开发;以及进一步提高基因治疗的疗效。