Brain pharmacology of intrathecal antisense oligonucleotides revealed through multimodal imaging

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 i...

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Bibliographic Details
Published in:JCI Insight
Main Authors: Mazur, Curt, Powers, Berit, Zasadny, Kenneth, Sullivan, Jenna M., Dimant, Hemi, Kamme, Fredrik, Hesterman, Jacob, Matson, John, Oestergaard, Michael, Seaman, Marc, Holt, Robert W., Qutaish, Mohammed, Polyak, Ildiko, Coelho, Richard, Gottumukkala, Vijay, Gaut, Carolynn M., Berridge, Marc, Albargothy, Nazira J., Kelly, Louise, Carare, Roxana O., Hoppin, Jack, Kordasiewicz, Holly, Swayze, Eric E., Verma, Ajay
Format: Artigo
Language:Inglês
Published: American Society for Clinical Investigation 2019
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Research Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6824309/
https://ncbi.nlm.nih.gov/pubmed/31619586
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.129240
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