Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing

Bowen Li; Rajith Singh Manan; Shun Qing Liang; Akiva Gordon; Allen Jiang; Andrew Varley; Guangping Gao; Robert Langer; Wen Xue; Daniel Anderson

doi:10.1038/s41587-023-01679-x

Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing

Bowen Li, Rajith Singh Manan, Shun Qing Liang, Akiva Gordon, Allen Jiang, Andrew Varley, Guangping Gao, Robert Langer, Wen Xue, Daniel Anderson

Research output: Contribution to journal › Article › peer-review

194 Scopus citations

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.

Original language	English (US)
Pages (from-to)	1410-1415
Number of pages	6
Journal	Nature biotechnology
Volume	41
Issue number	10
DOIs	https://doi.org/10.1038/s41587-023-01679-x
State	Published - Oct 2023
Externally published	Yes

Bibliographical note

Publisher Copyright:
© 2023, The Author(s), under exclusive licence to Springer Nature America, Inc.

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AU - Jiang, Allen

AU - Varley, Andrew

AU - Gao, Guangping

AU - Langer, Robert

AU - Xue, Wen

AU - Anderson, Daniel

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Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing

Abstract

Bibliographical note

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