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Opportunities and Challenges in the Delivery of mRNA-Based Vaccines

In the past few years, there has been increasing focus on the use of messenger RNA (mRNA) as a new therapeutic modality. Current clinical efforts encompassing mRNA-based drugs are directed toward infectious disease vaccines, cancer immunotherapies, therapeutic protein replacement therapies, and trea...

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Autores principales: Wadhwa, Abishek, Aljabbari, Anas, Lokras, Abhijeet, Foged, Camilla, Thakur, Aneesh
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7076378/
https://www.ncbi.nlm.nih.gov/pubmed/32013049
http://dx.doi.org/10.3390/pharmaceutics12020102
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author Wadhwa, Abishek
Aljabbari, Anas
Lokras, Abhijeet
Foged, Camilla
Thakur, Aneesh
author_facet Wadhwa, Abishek
Aljabbari, Anas
Lokras, Abhijeet
Foged, Camilla
Thakur, Aneesh
author_sort Wadhwa, Abishek
collection PubMed
description In the past few years, there has been increasing focus on the use of messenger RNA (mRNA) as a new therapeutic modality. Current clinical efforts encompassing mRNA-based drugs are directed toward infectious disease vaccines, cancer immunotherapies, therapeutic protein replacement therapies, and treatment of genetic diseases. However, challenges that impede the successful translation of these molecules into drugs are that (i) mRNA is a very large molecule, (ii) it is intrinsically unstable and prone to degradation by nucleases, and (iii) it activates the immune system. Although some of these challenges have been partially solved by means of chemical modification of the mRNA, intracellular delivery of mRNA still represents a major hurdle. The clinical translation of mRNA-based therapeutics requires delivery technologies that can ensure stabilization of mRNA under physiological conditions. Here, we (i) review opportunities and challenges in the delivery of mRNA-based therapeutics with a focus on non-viral delivery systems, (ii) present the clinical status of mRNA vaccines, and (iii) highlight perspectives on the future of this promising new type of medicine.
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spelling pubmed-70763782020-03-24 Opportunities and Challenges in the Delivery of mRNA-Based Vaccines Wadhwa, Abishek Aljabbari, Anas Lokras, Abhijeet Foged, Camilla Thakur, Aneesh Pharmaceutics Review In the past few years, there has been increasing focus on the use of messenger RNA (mRNA) as a new therapeutic modality. Current clinical efforts encompassing mRNA-based drugs are directed toward infectious disease vaccines, cancer immunotherapies, therapeutic protein replacement therapies, and treatment of genetic diseases. However, challenges that impede the successful translation of these molecules into drugs are that (i) mRNA is a very large molecule, (ii) it is intrinsically unstable and prone to degradation by nucleases, and (iii) it activates the immune system. Although some of these challenges have been partially solved by means of chemical modification of the mRNA, intracellular delivery of mRNA still represents a major hurdle. The clinical translation of mRNA-based therapeutics requires delivery technologies that can ensure stabilization of mRNA under physiological conditions. Here, we (i) review opportunities and challenges in the delivery of mRNA-based therapeutics with a focus on non-viral delivery systems, (ii) present the clinical status of mRNA vaccines, and (iii) highlight perspectives on the future of this promising new type of medicine. MDPI 2020-01-28 /pmc/articles/PMC7076378/ /pubmed/32013049 http://dx.doi.org/10.3390/pharmaceutics12020102 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Wadhwa, Abishek
Aljabbari, Anas
Lokras, Abhijeet
Foged, Camilla
Thakur, Aneesh
Opportunities and Challenges in the Delivery of mRNA-Based Vaccines
title Opportunities and Challenges in the Delivery of mRNA-Based Vaccines
title_full Opportunities and Challenges in the Delivery of mRNA-Based Vaccines
title_fullStr Opportunities and Challenges in the Delivery of mRNA-Based Vaccines
title_full_unstemmed Opportunities and Challenges in the Delivery of mRNA-Based Vaccines
title_short Opportunities and Challenges in the Delivery of mRNA-Based Vaccines
title_sort opportunities and challenges in the delivery of mrna-based vaccines
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7076378/
https://www.ncbi.nlm.nih.gov/pubmed/32013049
http://dx.doi.org/10.3390/pharmaceutics12020102
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