Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. Implications for gene therapy in humans.
MT: delivery strategies
autosomal dominant osteopetrosis
bone resorption
delivery nanoparticles
genetic bone diseases
osteoclasts
silicon
therapeutic siRNA
therapy
Journal
Molecular therapy. Nucleic acids
ISSN: 2162-2531
Titre abrégé: Mol Ther Nucleic Acids
Pays: United States
ID NLM: 101581621
Informations de publication
Date de publication:
12 Sep 2023
12 Sep 2023
Historique:
received:
25
03
2023
accepted:
16
08
2023
medline:
8
9
2023
pubmed:
8
9
2023
entrez:
8
9
2023
Statut:
epublish
Résumé
Rare skeletal diseases are still in need of proper clinically available transfection agents as the major challenge for first-in-human translation relates to intrinsic difficulty in targeting bone without exacerbating any inherent toxicity due to used vector. SiSaf's silicon stabilized hybrid lipid nanoparticles (sshLNPs) constitute next-generation non-viral vectors able to retain the integrity and stability of constructs and to accommodate considerable payloads of biologicals, without requiring cold-chain storage. sshLNP was complexed with a small interfering RNA (siRNA) specifically designed against the human
Identifiants
pubmed: 37680985
doi: 10.1016/j.omtn.2023.08.020
pii: S2162-2531(23)00231-7
pmc: PMC10480457
doi:
Types de publication
Journal Article
Langues
eng
Pagination
925-937Informations de copyright
© 2023 The Author(s).
Déclaration de conflit d'intérêts
A.T. and A.M. served as consultant, P.P had a service agreement, and F.M.S., N.T.-P., P.B.R, C.B., U.N., M.W., and A.D. are scientists working for SiSaf Ltd, a commercial stage biopharmaceutical company. S.S.-S. is founder and CEO of SiSaf Ltd.
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