PEPTIDE LIGANDS FOR CNS AND OCULAR DELIVERY OF RNAI COMPOUNDS
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United States of America
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Mar 6, 2025
app pub date -
Jul 29, 2022
filing date -
Jul 29, 2022
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Abstract
The disclosure relates to RNA agents targeting LRP1 receptor modified for targeted delivery to the brain and/or the eye. The present invention provides modified double stranded ribonucleic acid (dsRNAi) agents conjugated to a peptide ligand, as well as methods of modulating the expression of a target gene in a CNS cell or tissue and/or an ocular cell or tissue and methods of treating subjects having a CNS and/or an ocular disease or disorder using such dsRNAi agents.
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