Department of Cardiothoracic Surgery, Changzheng Hospital, Second Military Medical University, Fengyang Road 415#, Shanghai 200003, China.
Department of Cardiovascular Surgery, Institute of Cardiac Surgery, PLA General Hospital, Beijing, China.
Curr Top Med Chem. 2019;19(27):2507-2523. doi: 10.2174/1568026619666191024170212.
The rapid development of nanotechnology has a great influence on the fields of biology, physiology, and medicine. Over recent years, nanoparticles have been widely presented as nanocarriers to help the delivery of gene, drugs, and other therapeutic agents with cellular targeting ability. Advances in the understanding of gene delivery and RNA interference (RNAi)-based therapy have brought increasing attention to understanding and tackling complex genetically related diseases, such as cancer, cardiovascular and pulmonary diseases, autoimmune diseases and infections. The combination of nanocarriers and DNA/RNA delivery may potentially improve their safety and therapeutic efficacy. However, there still exist many challenges before this approach can be practiced in the clinic. In this review, we provide a comprehensive summary on the types of nanoparticle systems used as nanocarriers, highlight the current use of nanocarriers in recombinant DNA and RNAi molecules delivery, and the current landscape of gene-based nanomedicine-ranging from diagnosis to therapeutics. Finally, we briefly discuss the biosafety concerns and limitations in the preclinical and clinical development of nanoparticle gene systems.
纳米技术的快速发展对生物学、生理学和医学领域产生了巨大的影响。近年来,纳米颗粒已被广泛用作纳米载体,以帮助具有细胞靶向能力的基因、药物和其他治疗剂的传递。对基因传递和基于 RNA 干扰 (RNAi) 的治疗的理解的进步引起了人们对复杂遗传相关疾病(如癌症、心血管和肺部疾病、自身免疫性疾病和感染)的认识和治疗的日益关注。纳米载体与 DNA/RNA 传递的结合可能会提高它们的安全性和治疗效果。然而,在这种方法能够在临床上实施之前,仍然存在许多挑战。在这篇综述中,我们全面总结了用作纳米载体的纳米颗粒系统的类型,重点介绍了纳米载体在重组 DNA 和 RNAi 分子传递中的当前用途,以及基因纳米医学的现状——从诊断到治疗。最后,我们简要讨论了纳米颗粒基因系统在临床前和临床开发中的生物安全问题和局限性。