NeuroscienceNucleic Acid TherapeuticsPeer-reviewed
Cell type distribution of intrathecal antisense oligonucleotide activity in deep brain regions of non-human primates
Jeannine A. Frei, Juliana E Gentile, Yuan Lian…·Eric Vallabh Minikel
The paper assesses the cell-type-specific activity of intrathecally delivered antisense oligonucleotides in deep brain regions of non-human primates, finding broad target engagement across neuronal and non-neuronal cells.