| dc.contributor.author | Li, Bowen | |
| dc.contributor.author | Manan, Rajith Singh | |
| dc.contributor.author | Liang, Shun-Qing | |
| dc.contributor.author | Gordon, Akiva | |
| dc.contributor.author | Jiang, Allen | |
| dc.contributor.author | Varley, Andrew | |
| dc.contributor.author | Gao, Guangping | |
| dc.contributor.author | Langer, Robert | |
| dc.contributor.author | Xue, Wen | |
| dc.contributor.author | Anderson, Daniel | |
| dc.date.accessioned | 2024-09-18T20:42:06Z | |
| dc.date.available | 2024-09-18T20:42:06Z | |
| dc.date.issued | 2023-10 | |
| dc.identifier.uri | https://hdl.handle.net/1721.1/156898 | |
| dc.description.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. | en_US |
| dc.language.iso | en | |
| dc.publisher | Springer Science and Business Media LLC | en_US |
| dc.relation.isversionof | 10.1038/s41587-023-01679-x | en_US |
| dc.rights | Creative Commons Attribution-Noncommercial-ShareAlike | en_US |
| dc.rights.uri | http://creativecommons.org/licenses/by-nc-sa/4.0/ | en_US |
| dc.source | PubMed Central | en_US |
| dc.title | Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing | en_US |
| dc.type | Article | en_US |
| dc.identifier.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). | en_US |
| dc.contributor.department | Koch Institute for Integrative Cancer Research at MIT | en_US |
| dc.contributor.department | Massachusetts Institute of Technology. Department of Chemical Engineering | en_US |
| dc.contributor.department | Massachusetts Institute of Technology. Institute for Medical Engineering & Science | en_US |
| dc.contributor.department | Harvard-MIT Program in Health Sciences and Technology | en_US |
| dc.relation.journal | Nature Biotechnology | en_US |
| dc.eprint.version | Author's final manuscript | en_US |
| dc.type.uri | http://purl.org/eprint/type/JournalArticle | en_US |
| eprint.status | http://purl.org/eprint/status/PeerReviewed | en_US |
| dc.date.updated | 2024-09-18T20:29:04Z | |
| dspace.orderedauthors | Li, B; Manan, RS; Liang, S-Q; Gordon, A; Jiang, A; Varley, A; Gao, G; Langer, R; Xue, W; Anderson, D | en_US |
| dspace.date.submission | 2024-09-18T20:29:06Z | |
| mit.journal.volume | 41 | en_US |
| mit.journal.issue | 10 | en_US |
| mit.license | OPEN_ACCESS_POLICY | |
| mit.metadata.status | Authority Work and Publication Information Needed | en_US |
