1. Segmented poly(A) tails with microRNA target sites confer tissue-specific regulation for mRNA therapeutics
Qi R, Chen H, Chen R, Bao H, Sun Y, Xu R, Et Al.
Targeted delivery and controlled expression of mRNA encapsulated by lipid nanoparticles (mRNA-LNPs) are critical for the development of safe and effective mRNA medicines. However, efficient post-delivery regulation of mRNA-LNP expression remains challenging, and conventional approaches largely rely on modifying the 3' UTR at the expense of other regulatory elements. In this study, we engineered segmented poly(A) tail variants that function as gene-specific regulatory elements for synthetic mRNAs, providing an alternative regulatory module that preserves UTR integrity.
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