Article

Modulation of <i>TTR</i> Gene Expression in the Eye using Modified Duplex RNAs

2025-03-13

Abstract excerpt

Small interfering RNAs (siRNAs) are a proven therapeutic approach for controlling gene expression in the liver. Expanding the clinical potential of RNA interference (RNAi) requires developing strategies to enhance delivery to extra-hepatic tissues. In this study we examine inhibiting transthyretin ( TRR ) gene expression by short interfering RNAs (siRNAs) in the eye. Anti- TTR siRNAs have been developed as succ...

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Literature Corpus work: 0ed204ba-4a1d-5ce4-815e-5e900c92078a
DOI: 10.1101/2025.03.11.642595

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