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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Bibliografiska uppgifter
I publikationen:JCI Insight
Huvudupphovsmän: 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
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society for Clinical Investigation 2019
Ämnen:
Research Article
Länkar: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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