Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing.
Nat Biotechnol
; 41(10): 1410-1415, 2023 Oct.
Article
en En
| MEDLINE
| ID: mdl-36997680
ABSTRACT
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.
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Banco de datos:
MEDLINE
Idioma:
En
Revista:
Nat Biotechnol
Asunto de la revista:
BIOTECNOLOGIA
Año:
2023
Tipo del documento:
Article
País de afiliación:
Estados Unidos