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

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...

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Autores principales: Ylösmäki, Leena, Polini, Beatrice, Carpi, Sara, Martins, Beatriz, Smertina, Elena, Feola, Sara, Fusciello, Manlio, Peltonen, Karita, Nieri, Paola, Ylösmäki, Erkko, Cerullo, Vincenzo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6808555/
https://www.ncbi.nlm.nih.gov/pubmed/31644552
http://dx.doi.org/10.1371/journal.pone.0224072
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author Ylösmäki, Leena
Polini, Beatrice
Carpi, Sara
Martins, Beatriz
Smertina, Elena
Feola, Sara
Fusciello, Manlio
Peltonen, Karita
Nieri, Paola
Ylösmäki, Erkko
Cerullo, Vincenzo
author_facet Ylösmäki, Leena
Polini, Beatrice
Carpi, Sara
Martins, Beatriz
Smertina, Elena
Feola, Sara
Fusciello, Manlio
Peltonen, Karita
Nieri, Paola
Ylösmäki, Erkko
Cerullo, Vincenzo
author_sort Ylösmäki, Leena
collection PubMed
description 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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spelling pubmed-68085552019-11-02 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 PLoS One Research Article 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. Public Library of Science 2019-10-23 /pmc/articles/PMC6808555/ /pubmed/31644552 http://dx.doi.org/10.1371/journal.pone.0224072 Text en © 2019 Ylösmäki et al http://creativecommons.org/licenses/by/4.0/ This is an open access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/) , which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
spellingShingle Research Article
Ylösmäki, Leena
Polini, Beatrice
Carpi, Sara
Martins, Beatriz
Smertina, Elena
Feola, Sara
Fusciello, Manlio
Peltonen, Karita
Nieri, Paola
Ylösmäki, Erkko
Cerullo, Vincenzo
Harnessing therapeutic viruses as a delivery vehicle for RNA-based therapy
title Harnessing therapeutic viruses as a delivery vehicle for RNA-based therapy
title_full Harnessing therapeutic viruses as a delivery vehicle for RNA-based therapy
title_fullStr Harnessing therapeutic viruses as a delivery vehicle for RNA-based therapy
title_full_unstemmed Harnessing therapeutic viruses as a delivery vehicle for RNA-based therapy
title_short Harnessing therapeutic viruses as a delivery vehicle for RNA-based therapy
title_sort harnessing therapeutic viruses as a delivery vehicle for rna-based therapy
topic Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6808555/
https://www.ncbi.nlm.nih.gov/pubmed/31644552
http://dx.doi.org/10.1371/journal.pone.0224072
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