A single-cell map of antisense oligonucleotide activity in the brain
Meredith A Mortberg, Juliana E Gentile, Naeem M. Nadaf, Charles R. Vanderburg, Sean Simmons, Dan Dubinsky, Adam Slamin, Salome Maldonado, Caroline L Petersen, Nichole Jones, Holly B. Kordasiewicz, Hien Tran Zhao, Sonia M. Vallabh, Eric Vallabh Minikel
The paper maps antisense oligonucleotide activity in the brain using single-cell transcriptomics in mice and macaques, showing widespread target suppression across cell types with varying durations.
Nucleic Acids Research · May 15, 2023Peer-reviewedNeuroscience