Home Products Cited in Publications Worldwide Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing
Nat. Biotechnol.,2023,41(10):1410-1415.
Li, Bowen; Manan, Rajith Singh; Liang, Shun-Qing; Gordon, Akiva; Jiang, Allen; Varley, Andrew; Gao, Guangping; Langer, Robert; Xue, Wen; Anderson, Daniel
DOI:10.1038/s41587-023-01679-x PMID:36997680
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 mRNA 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.