治疗性 RNA 递送至 COVID 和其他疾病。
Therapeutic RNA Delivery for COVID and Other Diseases.
机构信息
Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine, Atlanta, GA, 30332, USA.
出版信息
Adv Healthc Mater. 2021 Aug;10(15):e2002022. doi: 10.1002/adhm.202002022. Epub 2021 Mar 4.
RNA can alter the expression of endogenous genes and can be used to express therapeutic proteins. As a result, RNA-based therapies have recently mitigated disease in patients. Yet most potential RNA therapies cannot currently be developed, in large part because delivering therapeutic quantities of RNA drugs to diseased cells remains difficult. Here, recent studies focused on the biological hurdles that make in vivo drug delivery challenging are described. Then RNA drugs that have overcome these challenges in humans, focusing on siRNA to treat liver disease and mRNA to vaccinate against COVID, are discussed. Finally, research centered on improving drug delivery to new tissues is highlighted, including the development of high-throughput in vivo nanoparticle DNA barcoding assays capable of testing over 100 distinct nanoparticles in a single animal.
RNA 可以改变内源性基因的表达，并且可以用来表达治疗性蛋白质。因此，基于 RNA 的疗法最近减轻了患者的疾病。然而，目前大多数潜在的 RNA 疗法都无法开发，很大程度上是因为向患病细胞输送治疗量的 RNA 药物仍然很困难。在这里，描述了最近集中研究使体内药物输送具有挑战性的生物学障碍的研究。然后讨论了在人类中克服这些挑战的 RNA 药物，重点是针对肝病的 siRNA 和针对 COVID 的 mRNA 疫苗。最后，突出了以改善向新组织输送药物为中心的研究，包括开发高通量体内纳米颗粒 DNA 条码检测，该检测能够在单个动物中测试超过 100 种不同的纳米颗粒。
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