Engineering poly- and micelleplexes for nucleic acid delivery - A reflection on their endosomal escape.
Nonviral vector
Polyethyleneimine
Proton sponge hypothesis
RNA
Vaccine
Journal
Journal of controlled release : official journal of the Controlled Release Society
ISSN: 1873-4995
Titre abrégé: J Control Release
Pays: Netherlands
ID NLM: 8607908
Informations de publication
Date de publication:
01 2023
01 2023
Historique:
received:
10
10
2022
revised:
02
12
2022
accepted:
03
12
2022
pubmed:
11
12
2022
medline:
3
2
2023
entrez:
10
12
2022
Statut:
ppublish
Résumé
For the longest time, the field of nucleic acid delivery has remained skeptical whether or not polycationic drug carrier systems would ever make it into clinical practice. Yet, with the disclosure of patents on polyethyleneimine-based RNA carriers through leading companies in the field of nucleic acid therapeutics such as BioNTech SE and the progress in clinical studies beyond phase I trials, this aloofness seems to regress. As one of the most striking characteristics of polymer-based vectors, the extraordinary tunability can be both a blessing and a curse. Yet, knowing about the adjustment screws and how they impact the performance of the drug carrier provides the formulation scientist committed to its development with a head start. Here, we equip the reader with a toolbox - a toolbox that should advise and support the developer to conceptualize a cutting-edge poly- or micelleplex system for the delivery of therapeutic nucleic acids; to be specific, to engineer the vector towards maximum endosomal escape performance at minimum toxicity. Therefore, after briefly sketching the boundary conditions of polymeric vector design, we will dive into the topic of endosomal trafficking. We will not only discuss the most recent knowledge of the endo-lysosomal compartment but further depict different hypotheses and mechanisms that facilitate the endosomal escape of polyplex systems. Finally, we will combine the different facets introduced in the previous chapters with the fundamental building blocks of polymer vector design and evaluate the advantages and drawbacks. Throughout the article, a particular focus will be placed on cellular peculiarities, not only as an additional barrier, but also to give inspiration to how such cell-specific traits might be capitalized on.
Identifiants
pubmed: 36496051
pii: S0168-3659(22)00822-7
doi: 10.1016/j.jconrel.2022.12.008
pmc: PMC9900387
pii:
doi:
Substances chimiques
Nucleic Acids
0
Drug Carriers
0
Polymers
0
RNA
63231-63-0
Types de publication
Journal Article
Review
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
518-534Informations de copyright
Copyright © 2022 The Authors. Published by Elsevier B.V. All rights reserved.
Déclaration de conflit d'intérêts
Declaration of Competing Interest The authors declare no competing interest.
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