Impact of the Core Chemistry of Self-Assembled Spherical Nucleic Acids on their In Vitro Fate.
Disulfide
Drug Delivery
Gene Silencing
Spherical Nucleic Acids
Stimuli-Responsive
Journal
Angewandte Chemie (International ed. in English)
ISSN: 1521-3773
Titre abrégé: Angew Chem Int Ed Engl
Pays: Germany
ID NLM: 0370543
Informations de publication
Date de publication:
18 Dec 2023
18 Dec 2023
Historique:
received:
18
10
2023
pubmed:
31
10
2023
medline:
31
10
2023
entrez:
31
10
2023
Statut:
ppublish
Résumé
Nucleic acid therapeutics (NATs), such as mRNA, small interfering RNA or antisense oligonucleotides are extremely efficient tools to modulate gene expression and tackle otherwise undruggable diseases. Spherical nucleic acids (SNAs) can efficiently deliver small NATs to cells while protecting their payload from nucleases, and have improved biodistribution and muted immune activation. Self-assembled SNAs have emerged as nanostructures made from a single DNA-polymer conjugate with similar favorable properties as well as small molecule encapsulation. However, because they maintain their structure by non-covalent interactions, they might suffer from disassembly in biologically relevant conditions, especially with regard to their interaction with serum proteins. Here, we report a systematic study of the factors that govern the fate of self-assembled SNAs. Varying the core chemistry and using stimuli-responsive disulfide crosslinking, we show that extracellular stability upon binding with serum proteins is important for recognition by membrane receptors, triggering cellular uptake. At the same time, intracellular dissociation is required for efficient therapeutic release. Disulfide-crosslinked SNAs combine these two properties and result in efficient and non-toxic unaided gene silencing therapeutics. We anticipate these investigations will help the translation of promising self-assembled structures towards in vivo gene silencing applications.
Identifiants
pubmed: 37905978
doi: 10.1002/anie.202315768
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e202315768Subventions
Organisme : Natural Sciences and Engineering Research Council
ID : RGPIN-2018-06861
Organisme : Canada Foundation for Innovation
ID : 23690
Organisme : Fonds de Recherche du Québec-Nature et technologies
ID : PR-284731
Organisme : Schweizerischer Nationalfonds zur Förderung der Wissenschaftlichen Forschung
ID : P500PN_206907
Informations de copyright
© 2023 The Authors. Angewandte Chemie International Edition published by Wiley-VCH GmbH.
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