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

• Main Authors: Rachael Miller (Author), Joseph Paquette (Author), Alexandra Barker (Author), Ellen Sapp (Author), Nicholas McHugh (Author), Brianna Bramato (Author), Nozomi Yamada (Author), Julia Alterman (Author), Dimas Echeveria (Author), Ken Yamada (Author), Jonathan Watts (Author), Christelle Anaclet (Author), Marian DiFiglia (Author), Anastasia Khvorova (Author), Neil Aronin (Author)
• Format: Book
• Published: Elsevier, 2024-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...