Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

mRNA has broad potential as a therapeutic. Current clinical efforts are focused on vaccination, protein replacement therapies, and treatment of genetic diseases. The clinical translation of mRNA therapeutics has been made possible through advances in the design of mRNA manufacturing and intracellula...

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Bibliographic Details
Published in:	Molecular therapy 2019-04, Vol.27 (4), p.710-728
Main Authors:	Kowalski, Piotr S., Rudra, Arnab, Miao, Lei, Anderson, Daniel G.
Format:	Article
Language:	eng
Subjects:	Animals Biomaterials Cell-Penetrating Peptides - chemistry clinical trial CRISPR CRISPR-Cas Systems Cytomegalovirus Dendrimers - chemistry Drug Delivery Systems - methods gene editing Gene Editing - methods gene therapy Genetic Therapy - methods Humans Immunization lipid nanoparticles Lipids Lipids - chemistry Mice mRNA mRNA delivery mRNA nanoparticles mRNA vaccine Nanoparticles - chemistry Polymers - chemistry protein replacement Review RNA, Messenger - genetics RNA, Messenger - therapeutic use Skin cancer Translation Vaccination Vaccination - methods Zika virus
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Summary:	mRNA has broad potential as a therapeutic. Current clinical efforts are focused on vaccination, protein replacement therapies, and treatment of genetic diseases. The clinical translation of mRNA therapeutics has been made possible through advances in the design of mRNA manufacturing and intracellular delivery methods. However, broad application of mRNA is still limited by the need for improved delivery systems. In this review, we discuss the challenges for clinical translation of mRNA-based therapeutics, with an emphasis on recent advances in biomaterials and delivery strategies, and we present an overview of the applications of mRNA-based delivery for protein therapy, gene editing, and vaccination. This review addresses the challenges for clinical translation of mRNA-based therapeutics, with an emphasis on recent advances in biomaterials and delivery strategies, and the authors present an overview of the applications of mRNA-based delivery for protein therapy, gene editing, and vaccination.
ISSN:	1525-0016 1525-0024