Programmable mRNA therapeutics for controlled epigenomic modulation of single and multiplexed gene expression in diverse diseases.

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Tác giả: Lauren Beech, Houda Belaghzal, Giuliana Castello Coatti, Justin Chen, Daniel F G Costa, Prachi Dhanania, Jeremiah D Farelli, Marcus I Gibson, Mayur Gurnani, Adam Katz, Thomas G McCauley, Samuel Mildrum, Chevaun Morrison-Smith, Joseph V Newman, Charles W O'Donnell, Pranjali Rumale, Stephen Siecinski, James Sullivan, Yaoyu E Wang, Caitlyn R Webb

Ngôn ngữ: eng

Ký hiệu phân loại: 599.073 Collections of living mammals

Thông tin xuất bản: England : Nature communications , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 711435

Pathogenic gene dysregulation can be attributed to chromatin state change that pre-transcriptionally regulates expression. Recent breakthroughs elucidating the rules governing this DNA control layer, an epigenetic code, unlock a modality in precision medicine to target gene dysregulation across myriad diseases. Here we present a modular platform to design programmable mRNA therapeutics, Epigenomic Controllers (EC), that control gene expression through directed epigenetic change. By leveraging natural mechanisms, ECs tune expression levels of one or multiple genes with durable effect of weeks-to-months in female mice following a single dose. We design and characterize ECs to multiple target genes and identify an EC that effectively inhibits the cancer- and inflammatory-disorder-associated multi-gene cluster CXCL1-8. With precision targeting of NF-kB signaling and identification of homologous murine surrogates, ECs significantly reduce neutrophil migration in vivo during acute lung inflammation in female mice. A platform approach to EC design for epigenomic modulation expands treatment frontiers for diverse gene targets, including those considered "undruggable."
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