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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Hlavní autoři: Flavien Bizot, Abdallah Fayssoil, Cécile Gastaldi, Tabitha Irawan, Xaysongkhame Phongsavanh, Arnaud Mansart, Thomas Tensorer, Elise Brisebard, Luis Garcia, Rudolph L Juliano, Aurélie Goyenvalle
Médium: Článek
Jazyk:English
Vydáno: MDPI AG 2023-02-01
Edice:Cells
Témata:
antisense oligonucleotides
exon-skipping
endosomal escape
duchenne muscular dystrophy
UNC7938
tricyclo-DNA
On-line přístup:https://www.mdpi.com/2073-4409/12/5/702

Internet

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

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