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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Detaylı Bibliyografya
Asıl Yazarlar: Flavien Bizot, Abdallah Fayssoil, Cécile Gastaldi, Tabitha Irawan, Xaysongkhame Phongsavanh, Arnaud Mansart, Thomas Tensorer, Elise Brisebard, Luis Garcia, Rudolph L Juliano, Aurélie Goyenvalle
Materyal Türü: Makale
Dil:English
Baskı/Yayın Bilgisi: MDPI AG 2023-02-01
Seri Bilgileri:Cells
Konular:
antisense oligonucleotides
exon-skipping
endosomal escape
duchenne muscular dystrophy
UNC7938
tricyclo-DNA
Online Erişim:https://www.mdpi.com/2073-4409/12/5/702

Internet

https://www.mdpi.com/2073-4409/12/5/702

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