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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Главные авторы:	Michaella Georgiadou, Melina Christou, Kleitos Sokratous, Jesper Wengel, Kyriaki Michailidou, Kyriacos Kyriacou, Andrie Koutsoulidou, Nikolaos P. Mastroyiannopoulos, Leonidas A. Phylactou
Формат:	Artigo
Язык:	Inglês
Опубликовано:	MDPI AG 2021-10-01
Серии:	Pharmaceuticals
Предметы:	DMD exon skipping antisense oligonucleotides LNA/2′<i>O</i>Me mdx
Online-ссылка:	https://www.mdpi.com/1424-8247/14/11/1113
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