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

Leena Ylösmäki; Beatrice Polini; Sara Carpi; Beatriz Martins; Elena Smertina; Sara Feola; Manlio Fusciello; Karita Peltonen; Paola Nieri; Erkko Ylösmäki; Vincenzo Cerullo

doi:10.1371/journal.pone.0224072

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

Leena Ylösmäki, Beatrice Polini, Sara Carpi, Beatriz Martins, Elena Smertina, Sara Feola, Manlio Fusciello, Karita Peltonen, Paola Nieri, Erkko Ylösmäki, Vincenzo Cerullo

Research output: Contribution to journal › Article › Scientific › peer-review

Abstract

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.

Original language	English
Journal	PLoS One
Volume	14
Issue number	10
Pages (from-to)	e0224072
ISSN	1932-6203
DOIs	https://doi.org/10.1371/journal.pone.0224072
Publication status	Published - 23 Oct 2019
MoE publication type	A1 Journal article-refereed

Fields of Science

317 Pharmacy
318 Medical biotechnology

Cite this

Ylösmäki, L., Polini, B., Carpi, S., Martins, B., Smertina, E., Feola, S., ... Cerullo, V. (2019). Harnessing therapeutic viruses as a delivery vehicle for RNA-based therapy. PLoS One, 14(10), e0224072. https://doi.org/10.1371/journal.pone.0224072

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abstract = "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.",

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Harnessing therapeutic viruses as a delivery vehicle for RNA-based therapy. / Ylösmäki, Leena; Polini, Beatrice; Carpi, Sara; Martins, Beatriz; Smertina, Elena; Feola, Sara ; Fusciello, Manlio ; Peltonen, Karita; Nieri, Paola; Ylösmäki, Erkko ; Cerullo, Vincenzo.

In: PLoS One, Vol. 14, No. 10, 23.10.2019, p. e0224072.

Research output: Contribution to journal › Article › Scientific › peer-review

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AU - Ylösmäki, Leena

AU - Polini, Beatrice

AU - Carpi, Sara

AU - Martins, Beatriz

AU - Smertina, Elena

AU - Feola, Sara

AU - Fusciello, Manlio

AU - Peltonen, Karita

AU - Nieri, Paola

AU - Ylösmäki, Erkko

AU - Cerullo, Vincenzo

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Access to Document

10.1371/journal.pone.0224072

journal.pone.0224072Final published version, 2.24 MBLicence: CC BY