Analysis of Exon Skipping Applicability for Dysferlinopathies

Analysis of Exon Skipping Applicability for Dysferlinopathies

Exon skipping, mediated through antisense oligonucleotides (ASOs), is a promising approach to exclude pathogenic variants from the <i>DYSF</i> gene and treat dysferlinopathies. Understanding the applicability of various exon skipping strategies in the total patient population, an analysi...

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Bibliographic Details
Main Authors: Jamie Leckie, Sebastian Hernandez Rodriguez, Martin Krahn, Toshifumi Yokota
Format: Article
Language:English
Published: MDPI AG 2025-01-01
Series:Cells
Subjects:
dysferlinopathy
exon skipping
antisense oligonucleotide (ASO)
applicability
Online Access:https://www.mdpi.com/2073-4409/14/3/177

Internet

https://www.mdpi.com/2073-4409/14/3/177

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