Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing.
Journal
Nature biotechnology
ISSN: 1546-1696
Titre abrégé: Nat Biotechnol
Pays: United States
ID NLM: 9604648
Informations de publication
Date de publication:
Oct 2023
Oct 2023
Historique:
received:
21
07
2022
accepted:
18
01
2023
pubmed:
31
3
2023
medline:
31
3
2023
entrez:
30
3
2023
Statut:
ppublish
Résumé
The expanding applications of nonviral genomic medicines in the lung remain restricted by delivery challenges. Here, leveraging a high-throughput platform, we synthesize and screen a combinatorial library of biodegradable ionizable lipids to build inhalable delivery vehicles for messenger RNA and CRISPR-Cas9 gene editors. Lead lipid nanoparticles are amenable for repeated intratracheal dosing and could achieve efficient gene editing in lung epithelium, providing avenues for gene therapy of congenital lung diseases.
Identifiants
pubmed: 36997680
doi: 10.1038/s41587-023-01679-x
pii: 10.1038/s41587-023-01679-x
pmc: PMC10544676
mid: NIHMS1924272
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1410-1415Subventions
Organisme : NHLBI NIH HHS
ID : DP2 HL137167
Pays : United States
Organisme : NHLBI NIH HHS
ID : P01 HL131471
Pays : United States
Organisme : NHLBI NIH HHS
ID : UG3 HL147367
Pays : United States
Organisme : NHLBI NIH HHS
ID : UH3 HL147367
Pays : United States
Informations de copyright
© 2023. The Author(s), under exclusive licence to Springer Nature America, Inc.
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