Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing
Name
nihms-1924272.pdf
Description
Published version
Size
1.42 MB
Format
Adobe PDF
Checksum (MD5)
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Author(s)
Li, Bowen
Manan, Rajith Singh
Liang, Shun-Qing
Gordon, Akiva
Jiang, Allen
Varley, Andrew
Gao, Guangping
Langer, Robert
Xue, Wen
Anderson, Daniel
Date Issued
October 2023
Journal
Nature Biotechnology
Publisher
Springer Science and Business Media LLC
Citation
Li, B., Manan, R.S., Liang, SQ. et al. Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing. Nat Biotechnol 41, 1410–1415 (2023).
Version
Author's final manuscript
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.
MIT Department
Koch Institute for Integrative Cancer Research at MIT
Massachusetts Institute of Technology. Department of Chemical Engineering
Massachusetts Institute of Technology. Institute for Medical Engineering & Science
Harvard-MIT Program in Health Sciences and Technology
Terms of Use
Creative Commons Attribution-Noncommercial-ShareAlike
Persistent DSpace Link
DOI of Published Version
https://doi.org/10.1038/s41587-023-01679-x
