Non-viral nucleic acid containing nanoparticles as cancer therapeutics.
Non-viral nucleic acid containing nanoparticles as cancer therapeutics.
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DOI：
10.1080/17425247.2016.1190707
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发表时间：
2016-10
影响因子：
6.6
通讯作者：
Green JJ
中科院分区：
文献类型：
--
作者：
Kozielski KL;Rui Y;Green JJ
核酸如DNA和短干扰RNA（siRNA）的递送通过实现新的生物学作用机制而有望用于治疗许多疾病，包括癌症。非病毒纳米颗粒是一类很有前途的核酸载体，可以设计成比传统病毒载体更安全和更通用。在这篇综述中，最近的进展，在细胞内交付的DNA和siRNA的描述与非病毒纳米粒子为基础的交付方法的重点。材料的性能，使成功的交付进行了讨论，以及已直接应用于癌症治疗的应用程序。共同递送不同核酸的策略被突出显示，核酸共同递送的新靶标也是如此。复杂的基于遗传的疾病如癌症的治疗可以通过多种核酸的安全和有效的细胞内递送来实现。可以制造非病毒纳米颗粒以将多种核酸同时递送到同一细胞，以防止肿瘤细胞容易地补偿一种遗传靶标的敲低或过表达。新的治疗方式和非病毒纳米技术的持续创新，以提供靶向特异性和个性化的基因治疗形式，为遗传医学在临床上治疗癌症等疾病带来了希望。
The delivery of nucleic acids such as DNA and short interfering RNA (siRNA) is promising for the treatment of many diseases, including cancer, by enabling novel biological mechanisms of action. Non-viral nanoparticles are a promising class of nucleic acid carriers that can be designed to be safer and more versatile than traditional viral vectors. In this review, recent advances in the intracellular delivery of DNA and siRNA are described with a focus on non-viral nanoparticle-based delivery methods. Material properties that have enabled successful delivery are discussed as well as applications that have directly been applied to cancer therapy. Strategies to co-deliver different nucleic acids are highlighted, as are novel targets for nucleic acid co-delivery. The treatment of complex genetically-based diseases such as cancer can be enabled by safe and effective intracellular delivery of multiple nucleic acids. Non-viral nanoparticles can be fabricated to deliver multiple nucleic acids to the same cell simultaneously to prevent tumor cells from easily compensating for the knockdown or overexpression of one genetic target. The continued innovation of new therapeutic modalities and non-viral nanotechnologies to provide target-specific and personalized forms of gene therapy hold promise for genetic medicine to treat diseases like cancer in the clinic.