Guanine Base Modifications in Antisense Oligonucleotides Mitigate Acute Central Nervous System Toxicity

Key Points

• The aim is to determine if guanine base modifications in antisense oligonucleotides can lessen neurotoxicity associated with intrathecal administration.
• Examining the effects of guanine base modifications on ASOs
• Assessing neurotoxicity in CNS models
• Conducting intrathecal administration experiments
• Guanine base modifications significantly reduced neurotoxicity
• Modified ASOs showed improved safety profiles in CNS applications