This research investigates guanine base modifications in antisense oligonucleotides to reduce neurotoxicity in CNS applications, implying improved safety for therapeutic use.
Antisense oligonucleotides (ASOs) are recognized as promising therapeutic agents for central nervous system (CNS) diseases. However, neurotoxicity induced by ASOs via intrathecal administration poses a major limitation for clinical use....
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Katsuyama et al. (2025) studied this question.
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