Article

Cell type distribution of intrathecal antisense oligonucleotide activity in deep brain regions of non-human primates

2025-05-21

Abstract excerpt

Intrathecally administered RNase H1-active gapmer antisense oligonucleotides (ASOs) are promising therapeutics for brain diseases where lowering the expression of one target gene is expected to be therapeutically beneficial. Such ASOs are active, to varying degrees, across most or all cell types in the cortex and cerebellum of mouse and non-human primate (NHP), brain regions with substantial drug accumulation. Int...

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Literature Corpus work: c5a34f5d-df53-598b-89a2-5f42e456a341
DOI: 10.1101/2025.05.20.655163

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