Article

RAG-17: A Novel siRNA Conjugate Demonstrating Efficacy in Late-Stage Treatment of SOD1 <sup>G93A</sup> ALS mice

2023-11-23

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease characterized by rapid progression and high mortality. With genetic mutations, particularly in the SOD1 gene, playing a significant role in ALS pathogenesis, targeted therapies have become a primary focus. This study introduces RD-12500 (RAG-17), a novel siRNA-ACO (Accessory Oligonucleotide) conjugate designed to address the challenges...

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Literature Corpus work: 12e8f1a6-24d8-504a-a437-fb759959d21b
DOI: 10.1101/2023.11.23.568255

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