Article

A programmable dual-targeting di-valent siRNA scaffold supports potent multi-gene modulation in the central nervous system

2023-12-19

Abstract excerpt

<h4>ABSTRACT</h4> Di-valent short interfering RNA (siRNA) is a promising therapeutic modality that enables sequence-specific modulation of a single target gene in the central nervous system (CNS). To treat complex neurodegenerative disorders, where pathogenesis is driven by multiple genes or pathways, di-valent siRNA must be able to silence multiple target genes simultaneously. Here we present a framework for des...

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Literature Corpus work: e60a12fc-ce93-582d-a80e-db82df51acb0
DOI: 10.1101/2023.12.19.572404

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