Article

Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation

2024-06-08

Abstract excerpt

Oligonucleotide therapeutics (ASOs and siRNAs) have been explored for modulation of gene expression in the central nervous system (CNS), with several drugs approved and many in clinical evaluation. Administration of highly concentrated oligonucleotides to the CNS can induce acute neurotoxicity. We demonstrate that delivery of concentrated oligonucleotides to the CSF in awake mice induces acute toxicity, observable...

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Identifiers and source

Literature Corpus work: 3eca9443-8401-5afa-8d46-5a42c4f967dc
DOI: 10.1101/2024.06.06.597639

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