Nanoscale platforms for messenger RNA delivery.

Nanoscale platforms for messenger RNA delivery.
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DOI:
10.1002/wnan.1530
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发表时间:
2019-03
期刊:
Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology
影响因子:
--
通讯作者:
Dong Y
Dong Y
中科院分区:
其他
文献类型:
--
作者:
Li B;Zhang X;Dong Y

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在过去的几年中,信使RNA(mRNA)已经成为用于各种治疗应用的有前途的一类药物。一系列临床试验正在进行或将在不久的将来启动,用于治疗各种疾病。目前,基于mRNA的治疗方法在临床研究中主要集中在离体转染和局部给药。治疗相关mRNA的有效和安全递送仍然是其在人类中广泛应用的主要挑战之一。因此,迫切需要有效的运载系统来克服这一限制。近年来,许多纳米级生物材料已被构建用于mRNA递送,以保护mRNA免受细胞外降解并促进细胞摄取后的内体逃逸。纳米级平台扩大了基于mRNA的治疗方法的可行性,并使其在蛋白质替代疗法、癌症免疫疗法、治疗性疫苗、再生医学和基因组编辑方面的潜在应用成为可能。本文综述了用于mRNA递送的纳米级平台的最新进展、挑战和未来方向,包括脂质和脂质衍生的纳米颗粒、基于聚合物的纳米颗粒、蛋白质衍生物mRNA复合物和其他类型的纳米材料。
Messenger RNA (mRNA) has become a promising class of drugs for diverse therapeutic applications in the past few years. A series of clinical trials are ongoing or will be initiated in the near future for the treatment of a variety of diseases. Currently, mRNA-based therapeutics mainly focuses on ex vivo transfection and local administration in clinical studies. Efficient and safe delivery of therapeutically relevant mRNAs remains one of the major challenges for their broad applications in humans. Thus, effective delivery systems are urgently needed to overcome this limitation. In recent years, numerous nanoscale biomaterials have been constructed for mRNA delivery in order to protect mRNA from extracellular degradation and facilitate endosomal escape after cellular uptake. Nanoscale platforms have expanded the feasibility of mRNA-based therapeutics, and enabled its potential applications to protein replacement therapy, cancer immunotherapy, therapeutic vaccines, regenerative medicine, and genome editing. This review focuses on recent advances, challenges, and future directions in nanoscale platforms designed for mRNA delivery, including lipid and lipid-derived nanoparticles, polymer-based nanoparticles, protein derivatives mRNA complexes, and other types of nanomaterials.
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