A single-cell map of antisense oligonucleotide activity in the brain

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Antisense oligonucleotides (ASOs) dosed into cerebrospinal fluid (CSF) distribute broadly throughout the brain and hold the promise of treating myriad brain diseases by modulating RNA. CNS tissue is not routinely biopsied in living individuals, leading to reliance on CSF biomarkers to inform on drug...

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Detalles Bibliográficos
Autores principales: Mortberg, Meredith A, Gentile, Juliana E, Nadaf, Naeem, Vanderburg, Charles, Simmons, Sean, Dubinsky, Dan, Slamin, Adam, Maldonado, Salome, Petersen, Caroline L, Jones, Nichole, Kordasiewicz, Holly B, Zhao, Hien T, Vallabh, Sonia M, Minikel, Eric Vallabh
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9948956/
https://www.ncbi.nlm.nih.gov/pubmed/36824749
http://dx.doi.org/10.1101/2023.02.14.528473
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