Brain pharmacology of intrathecal antisense oligonucleotides revealed through multimodal imaging
Abstract
Intrathecal (IT) delivery and pharmacology of antisense oligonucleotides (ASOs) for the CNS have been successfully developed to treat spinal muscular atrophy. However, ASO pharmacokinetic (PK) and pharmacodynamic (PD) properties remain poorly understood in the IT compartment. We applied multimodal imaging techniques to elucidate the IT PK and PD of unlabeled, radioactively labeled, or fluorescently labeled ASOs targeting ubiquitously expressed...
Paper Details
Title
Brain pharmacology of intrathecal antisense oligonucleotides revealed through multimodal imaging
Published Date
Oct 17, 2019
Journal
Volume
4
Issue
20
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