Intramuscular Evaluation of Chimeric Locked Nucleic Acid/2′<i>O</i>Methyl-Modified Antisense Oligonucleotides for Targeted Exon 23 Skipping in Mdx Mice

Intramuscular Evaluation of Chimeric Locked Nucleic Acid/2′<i>O</i>Methyl-Modified Antisense Oligonucleotides for Targeted Exon 23 Skipping in Mdx Mice

Duchenne muscular dystrophy (DMD) is a fatal disorder characterised by progressive muscle wasting. It is caused by mutations in the dystrophin gene, which disrupt the open reading frame leading to the loss of functional dystrophin protein in muscle fibres. Antisense oligonucleotide (AON)-mediated sk...

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Xehetasun bibliografikoak
Egile Nagusiak: Michaella Georgiadou, Melina Christou, Kleitos Sokratous, Jesper Wengel, Kyriaki Michailidou, Kyriacos Kyriacou, Andrie Koutsoulidou, Nikolaos P. Mastroyiannopoulos, Leonidas A. Phylactou
Formatua: Artikulua
Hizkuntza:English
Argitaratua: MDPI AG 2021-10-01
Saila:Pharmaceuticals
Gaiak:
DMD
exon skipping
antisense oligonucleotides
LNA/2′<i>O</i>Me
mdx
Sarrera elektronikoa:https://www.mdpi.com/1424-8247/14/11/1113

Internet

https://www.mdpi.com/1424-8247/14/11/1113

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