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. 2024 Dec 12;36(1):102423. doi: 10.1016/j.omtn.2024.102423

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© 2024 The Author(s)

This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

PMC Copyright notice

In vitro delivery of FANCC LNPs to correct FA-deficient BM-derived HSPCs

(A) Transmitted images of Lin⁻ whole bone marrow (BM) from Fancc^−/− mutant mice in methylcellulose media after treatment with LNP and 20 nM mitomycin C (MMC). (B) Colony survival rates expressed as a percentage ratio of the number of colonies formed after exposure to MMC and of colonies formed without MMC treatment across a range of treatments. (C) Percentage ratios of the sum of colony spread area of colonies in (B). (D) Transmitted images of ex vivo expanded LT-HSCs from Fancc^−/− mice in methylcellulose media after treatment with LNP and 30 nM MMC. (E and F) Colony survival rates of Fancc^−/− or WT ex vivo expanded LT-HSCs after pre-treatment with 30 nM MMC and LNP^LFancc or LNP^CFancc. Data points represent biological replicates (p < 0.05).