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Nature Materials ( IF 41.2 ) Pub Date : 2024-04-03 , DOI: 10.1038/s41563-024-01859-3
Philip Ball

The mRNA platform is potentially fast and versatile for delivering new vaccines in response to emerging infectious agents, requiring only the identification of a suitably immunogenic peptide fragment4. Not only could this approach be vital in the future pandemics that many epidemiologists regard as inevitable, but it is already providing treatments for common infectious diseases. RNA therapeutics is not all about mRNA, however. Regulatory non-coding RNAs (those transcribed from the genome that do not encode proteins and are involved in gene regulation) could be used both as the targets and the active agents of drugs5, from very short (micro)RNAs6 to long non-coding RNAs7.

While some of these agents might be delivered ‘naked’, in general they require effective delivery vehicles, albeit with sometimes contrasting demands: a vaccine ‘wants’ to be noticed by the immune system, whereas typically drugs do not. The mRNA COVID-19 vaccines were delivered using nanoparticles made from ionizable lipids, as is the small interfering RNA in the liver-targeting drug Onpattro used to treat polyneuropathy, a hereditary form of amyloidosis that causes nerve damage. (RNA interference is the process by which mRNAs are targeted by complementary small RNAs and degraded before they can be translated.)



中文翻译:

特快专递

mRNA 平台具有潜在的快速和通用性,可用于针对新出现的感染因子提供新疫苗,只需要识别适当的免疫原性肽片段4。这种方法不仅在许多流行病学家认为不可避免的未来流行病中至关重要,而且已经为常见传染病提供了治疗方法。然而,RNA 疗法并不全是关于 mRNA。调节性非编码 RNA(从基因组转录而来,不编码蛋白质并参与基因调节的 RNA)既可以用作药物的靶标,也可以用作药物的活性剂5,从非常短的(微小)RNA 6到长的非编码RNA。编码RNA 7

虽然其中一些药物可能是“裸”递送的,但一般来说,它们需要有效的递送载体,尽管有时要求相反:疫苗“希望”被免疫系统注意到,而通常药物则不然。 mRNA COVID-19 疫苗是使用由可电离脂质制成的纳米颗粒来传递的,就像用于治疗多发性神经病(一种导致神经损伤的遗传性淀粉样变性病)的肝脏靶向药物 Onpattro 中的小干扰 RNA 一样。 (RNA 干扰是 mRNA 被互补小 RNA 靶向并在翻译之前被降解的过程。)

更新日期:2024-04-05
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