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

• Published in: bioRxiv
• Main Authors: Miller, Rachael, Paquette, Joseph, Barker, Alexandra, Sapp, Ellen, McHugh, Nicholas, Bramato, Brianna, Yamada, Nozomi, Alterman, Julia, Echeveria, Dimas, Yamada, Ken, Watts, Jonathan, Anaclet, Christelle, DiFiglia, Marian, Khvorova, Anastasia, Aronin, Neil
• Format: Journal Article Paper
• Language: English
• Published: Cold Spring Harbor Laboratory 08.06.2024
• Edition: 1.1
• Subjects: Neuroscience

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 d...