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Harnessing therapeutic viruses as a delivery vehicle for RNA-based therapy

机译:利用治疗病毒作为用于RNA的疗法的递送载体

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Messenger RNA (mRNA) and microRNA (miRNA)-based therapeutics have become attractive alternatives to DNA-based therapeutics due to recent advances in manufacture, scalability and cost. Also, RNA-based therapeutics are considered safe since there are no risk of inducing genomic changes as well as the potential adverse effects would be only temporary due to the transient nature of RNA-based therapeutics. However, efficient in vivo delivery of RNA-based therapeutics remains a challenge. We have developed a delivery platform for RNA-based therapeutics by exploiting the physicochemical properties of enveloped viruses. By physically attaching cationic liposome/RNA complexes onto the viral envelope of vaccinia virus, we were able to deliver mRNA, self-replicating RNA as well as miRNA inside target cells. Also, we showed that this platform, called viRNA platform, can efficiently deliver functional miRNA mimics into B16.OVA tumour in vivo .
机译:由于近期制造,可扩展性和成本的进展,信使RNA(mRNA)和MicroRNA(miRNA)和MicroRNA(miRNA)的治疗方法已成为DNA的治疗剂的有吸引力的替代品。 此外,基于RNA的治疗剂被认为是安全的,因为没有诱导基因组变化的风险以及由于基于RNA的治疗剂的瞬态性质,潜在的不利影响是暂时的。 然而,高效地提供基于RNA的治疗剂的递送仍然是一个挑战。 通过利用包膜病毒的物理化学性质,我们开发了一种基于RNA的治疗方法的交付平台。 通过物理地将阳离子脂质体/ RNA复合物放到痘苗病毒病毒的病毒包络上,我们能够递送mRNA,自复制RNA以及靶细胞内的miRNA。 此外,我们表明,该平台称为Virna平台,可以有效地将功能性miRNA模拟物递送到体内B16.ova肿瘤中。

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