Article

SCAD Delivery Platform: A Novel Approach for Efficient CNS and Extrahepatic Oligonucleotide Therapeutics

2024-10-29

Abstract excerpt

<h4>ABSTRACT</h4> Oligonucleotide therapeutics, including antisense oligonucleotides (ASOs) and duplex RNAs like siRNA, saRNA, and miRNA, hold immense potential for treating genetic and acquired diseases by modulating gene expression in a target-specific manner. However, effective delivery to extrahepatic tissues, particularly the central nervous system (CNS), remains a significant challenge. While GalNAc conjuga...

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Literature Corpus work: 47f30893-5495-5b31-8883-3e64be184f02
DOI: 10.1101/2024.10.29.620792

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