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Data availability
Illumina Sequencing data have been submitted to the Sequence Read Archive; these datasets are available under BioProject Accession no. PRJNA918691 (ref. 37). The authors declare that all other data supporting the findings of this study are available within the paper and its Supplementary Information files or on reasonable request. Plasmids are available from Addgene.
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Acknowledgements
This work was supported by Translate Bio and the National Institutes of Health (grant no. UG3HL147367). B.L., R.S.M., A.G. and A.J. were supported by Translate Bio. B.L. was supported by the Leslie Dan Faculty of Pharmacy startup fund, the Connaught Fund (no. 514681), the J. P. Bickell Foundation (grant no. 515159), the Canada Research Chairs Program (no. CRC-2022-00575), Canadian Institutes of Health Research (no. PJH-185722) and the Canada Foundation for Innovation John R. Evans Leaders Fund (no. 43711). A.V. was supported by the PRiME Postdoctoral Fellowship from the University of Toronto. S.P.L., G.G., W.X. and D.A. were supported by grants from the National Institutes of Health (nos. UG3HL147367 and UH3HL147367). W.X. was supported by grants from the National Institutes of Health (nos. DP2HL137167 and P01HL131471), the American Cancer Society (grant no. 129056-RSG-16-093) and the Cystic Fibrosis Foundation. We thank the Koch Institute Swanson Biotechnology Center for technical support, specifically the Animal Imaging & Preclinical Testing, Histology, Nanotechnology Materials and Microscopy core facilities.
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Competing interests
B.L., R.S.M., A.G. and D.A. have filed a patent (PCT/US2022/052314) for development of the described lipids. D.A. receives research funding from Translate Bio and is a Founder of Orna Therapeutics. R.L. is a cofounder of Moderna; he also serves on the board and has equity in Particles for Humanity. For a list of entities with which R.L. is, or has been, recently involved, compensated or uncompensated, see https://www.dropbox.com/s/yc3xqb5s8s94v7x/Rev%20Langer%20COI.pdf?dl=0. The other authors declare no competing interests.
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Li, B., Manan, R.S., Liang, SQ. et al. Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing.
Nat Biotechnol (2023). https://doi.org/10.1038/s41587-023-01679-x
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DOI: https://doi.org/10.1038/s41587-023-01679-x
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