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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Autors principals:	Amel Saoudi, Claire Fergus, Talia Gileadi, Federica Montanaro, Jennifer E. Morgan, Vincent P. Kelly, Thomas Tensorer, Luis Garcia, Cyrille Vaillend, Francesco Muntoni, Aurélie Goyenvalle
Format:	Artigo
Idioma:	Inglês
Publicat:	MDPI AG 2023-03-01
Col·lecció:	Cells
Matèries:	ASO-based therapy central nervous system intracerebroventricular injection intrathecal injection antisense oligonucleotides exon-skipping
Accés en línia:	https://www.mdpi.com/2073-4409/12/6/908
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