BiochemistrybioRxivPreprint
Modulation of TTR Gene Expression in the Eye using Modified Duplex RNAs
Jiaxin Hu, Xin Gong, Jayanta Kundu…·David R. Corey
This study shows lipid-modified siRNAs can inhibit TTR gene expression in the eye via intravitreal injection, suggesting a potential therapy for ocular TTR amyloidosis.
DOI 10.1101/2025.03.11.642595