Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models

Nucleic acid-based therapies have demonstrated great potential for the treatment of monogenetic diseases, including neurologic disorders. To date, regulatory approval has been received for a dozen antisense oligonucleotides (ASOs); however, these chemistries cannot readily cross the blood–brain barr...

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Detalles Bibliográficos
Autores principales: Saoudi, Amel, Fergus, Claire, Gileadi, Talia, Montanaro, Federica, Morgan, Jennifer E., Kelly, Vincent P., Tensorer, Thomas, Garcia, Luis, Vaillend, Cyrille, Muntoni, Francesco, Goyenvalle, Aurélie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10047648/
https://www.ncbi.nlm.nih.gov/pubmed/36980249
http://dx.doi.org/10.3390/cells12060908

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10047648/
https://www.ncbi.nlm.nih.gov/pubmed/36980249
http://dx.doi.org/10.3390/cells12060908

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