Species-specific gene expression manipulation in humanized livers of chimeric mice via siRNA-encapsulated lipid nanoparticle treatment

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Mouse-specific siRNA-encapsulated lipid nanoparticles effectively suppress residual mouse gene expression in PXB-mice, creating a more accurate humanized liver model with human-like pharmacokinetic profiles. Novelty: ClaimNovelty.METHODOLOGICAL. Consensus alignment: ConsensusAlignment.NEUTRAL.

Kazuto Yamazaki; Kenji Kubara; Go Sugahara; Hiroki Muto; Masae Yamamoto; Yuji Mano; Kaoru Mitsuhashi; Chihiro Yamasaki; Yuji Ishida; Chise Tateno; Yuta Suzuki

doi:10.1016/j.omtm.2025.101466

April 14, 2025Molecular Therapy — Methods & Clinical DevelopmentOpen Access

Species-specific gene expression manipulation in humanized livers of chimeric mice via siRNA-encapsulated lipid nanoparticle treatment

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Abstract

-warfarin PK profile in PXB-mice more similar to that in humans. Thus, mouse-specific siRNA-LNP is a simple system to control gene expression in the remaining mouse hepatocytes of PXB-mice and create more humanized and invaluable models based on PXB-mice.

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Yamazaki et al. (2025) studied this question.

synapsesocial.com/papers/6a226cb939da1a1e729114a2 https://doi.org/10.1016/j.omtm.2025.101466

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Species-specific gene expression manipulation in humanized livers of chimeric mice via siRNA-encapsulated lipid nanoparticle treatment

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Abstract

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Species-specific gene expression manipulation in humanized livers of chimeric mice via siRNA-encapsulated lipid nanoparticle treatment

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Abstract

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