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

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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书目详细资料
Main Authors: Flavien Bizot, Abdallah Fayssoil, Cécile Gastaldi, Tabitha Irawan, Xaysongkhame Phongsavanh, Arnaud Mansart, Thomas Tensorer, Elise Brisebard, Luis Garcia, Rudolph L Juliano, Aurélie Goyenvalle
格式: 文件
语言:English
出版: MDPI AG 2023-02-01
丛编:Cells
主题:
antisense oligonucleotides
exon-skipping
endosomal escape
duchenne muscular dystrophy
UNC7938
tricyclo-DNA
在线阅读:https://www.mdpi.com/2073-4409/12/5/702

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