Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation
Rachael Miller, Joseph D. Paquette, Alexandra Barker, Ellen Sapp, Nicholas McHugh, Brianna M. Bramato, Nozomi Yamada, Julia F. Alterman, Dimas Echeverria, Ken Yamada, Jonathan K. Watts, Christelle Anaclet, Marian DiFiglia, Anastasia Khvorova, Neil Aronin M. D
This study shows adding high concentrations of Ca2+ and Mg2+ to oligonucleotide formulations prevents acute neurotoxicity in CNS delivery without affecting efficacy.
Molecular Therapy — Nucleic Acids · Oct 15, 2024Peer-reviewedNeuroscience