DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration

Antisense oligonucleotide–mediated (AO-mediated) therapy is a promising strategy to treat several neurological diseases, including spinal muscular atrophy (SMA). However, limited delivery to the CNS with AOs administered intravenously or subcutaneously is a major challenge. Here, we demonstrate a si...

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Detalles Bibliográficos
Autores principales: Aslesh, Tejal, Erkut, Esra, Ren, Jun, Lim, Kenji Rowel Q., Woo, Stanley, Hatlevig, Susan, Moulton, Hong M., Gosgnach, Simon, Greer, John, Maruyama, Rika, Yokota, Toshifumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2023
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10077475/
https://www.ncbi.nlm.nih.gov/pubmed/36719755
http://dx.doi.org/10.1172/jci.insight.160516

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10077475/
https://www.ncbi.nlm.nih.gov/pubmed/36719755
http://dx.doi.org/10.1172/jci.insight.160516

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