Scherer F, Anton M, Schillinger U, Henke J, Bergemann C, Krüger A, Gänsbacher B, Plank C
Technische Universität München, Institute of Experimental Oncology, Munich, Germany.
Gene Ther. 2002 Jan;9(2):102-9. doi: 10.1038/sj.gt.3301624.
Low efficiencies of nonviral gene vectors, the receptor-dependent host tropism of adenoviral or low titers of retroviral vectors limit their utility in gene therapy. To overcome these deficiencies, we associated gene vectors with superparamagnetic nanoparticles and targeted gene delivery by application of a magnetic field. This potentiated the efficacy of any vector up to several hundred-fold, allowed reduction of the duration of gene delivery to minutes, extended the host tropism of adenoviral vectors to nonpermissive cells and compensated for low retroviral titer. More importantly, the high transduction efficiency observed in vitro was reproduced in vivo with magnetic field-guided local transfection in the gastrointestinal tract and in blood vessels. Magnetofection provides a novel tool for high throughput gene screening in vitro and can help to overcome fundamental limitations to gene therapy in vivo.
非病毒基因载体的低效率、腺病毒依赖受体的宿主嗜性或逆转录病毒载体的低滴度限制了它们在基因治疗中的应用。为了克服这些缺陷，我们将基因载体与超顺磁性纳米颗粒相结合，并通过施加磁场实现靶向基因递送。这使任何载体的功效提高了数百倍，能够将基因递送的持续时间缩短至几分钟，将腺病毒载体的宿主嗜性扩展到非允许细胞，并弥补了逆转录病毒滴度低的问题。更重要的是，在体外观察到的高转导效率在体内通过胃肠道和血管中的磁场引导局部转染得以重现。磁转染为体外高通量基因筛选提供了一种新工具，并有助于克服体内基因治疗的基本限制。
