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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Prif Awduron:	Amel Saoudi, Claire Fergus, Talia Gileadi, Federica Montanaro, Jennifer E. Morgan, Vincent P. Kelly, Thomas Tensorer, Luis Garcia, Cyrille Vaillend, Francesco Muntoni, Aurélie Goyenvalle
Fformat:	Artigo
Iaith:	Inglês
Cyhoeddwyd:	MDPI AG 2023-03-01
Cyfres:	Cells
Pynciau:	ASO-based therapy central nervous system intracerebroventricular injection intrathecal injection antisense oligonucleotides exon-skipping
Mynediad Ar-lein:	https://www.mdpi.com/2073-4409/12/6/908
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