Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing
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 CR...
| Main Authors: | , , , , , , , , , |
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| Other Authors: | |
| Format: | Article |
| Language: | English |
| Published: |
Springer Science and Business Media LLC
2024
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| Online Access: | https://hdl.handle.net/1721.1/156898 |
| _version_ | 1824458223263940608 |
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| author | Li, Bowen Manan, Rajith Singh Liang, Shun-Qing Gordon, Akiva Jiang, Allen Varley, Andrew Gao, Guangping Langer, Robert Xue, Wen Anderson, Daniel |
| author2 | Koch Institute for Integrative Cancer Research at MIT |
| author_facet | Koch Institute for Integrative Cancer Research at MIT Li, Bowen Manan, Rajith Singh Liang, Shun-Qing Gordon, Akiva Jiang, Allen Varley, Andrew Gao, Guangping Langer, Robert Xue, Wen Anderson, Daniel |
| author_sort | Li, Bowen |
| collection | MIT |
| description | 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. |
| first_indexed | 2024-09-23T13:24:31Z |
| format | Article |
| id | mit-1721.1/156898 |
| institution | Massachusetts Institute of Technology |
| language | English |
| last_indexed | 2025-02-19T04:22:28Z |
| publishDate | 2024 |
| publisher | Springer Science and Business Media LLC |
| record_format | dspace |
| spelling | mit-1721.1/1568982025-01-04T04:43:07Z Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing Li, Bowen Manan, Rajith Singh Liang, Shun-Qing Gordon, Akiva Jiang, Allen Varley, Andrew Gao, Guangping Langer, Robert Xue, Wen Anderson, Daniel 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 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. 2024-09-18T20:42:06Z 2024-09-18T20:42:06Z 2023-10 2024-09-18T20:29:04Z Article http://purl.org/eprint/type/JournalArticle https://hdl.handle.net/1721.1/156898 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 10.1038/s41587-023-01679-x Nature Biotechnology Creative Commons Attribution-Noncommercial-ShareAlike http://creativecommons.org/licenses/by-nc-sa/4.0/ application/pdf Springer Science and Business Media LLC PubMed Central |
| spellingShingle | Li, Bowen Manan, Rajith Singh Liang, Shun-Qing Gordon, Akiva Jiang, Allen Varley, Andrew Gao, Guangping Langer, Robert Xue, Wen Anderson, Daniel Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing |
| title | Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing |
| title_full | Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing |
| title_fullStr | Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing |
| title_full_unstemmed | Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing |
| title_short | Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing |
| title_sort | combinatorial design of nanoparticles for pulmonary mrna delivery and genome editing |
| url | https://hdl.handle.net/1721.1/156898 |
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