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Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model

Nucleic acid-based therapeutics hold great promise for the treatment of numerous diseases, including neuromuscular disorders, such as Duchenne muscular dystrophy (DMD). Some antisense oligonucleotide (ASO) drugs have already been approved by the US FDA for DMD, but the potential of this therapy is s...

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Detalles Bibliográficos
Autores principales:	Bizot, Flavien, Fayssoil, Abdallah, Gastaldi, Cécile, Irawan, Tabitha, Phongsavanh, Xaysongkhame, Mansart, Arnaud, Tensorer, Thomas, Brisebard, Elise, Garcia, Luis, Juliano, Rudolph L, Goyenvalle, Aurélie
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10001065/ https://www.ncbi.nlm.nih.gov/pubmed/36899837 http://dx.doi.org/10.3390/cells12050702

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