Detailed genetic and functional analysis of the hDMDdel52/mdx mouse model.
Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disorder caused by reading frame disrupting mutations in the DMD gene leading to absence of functional dystrophin. Antisense oligonucleotide (AON)-mediated exon skipping is a therapeutic approach aimed at restoring the reading...
Main Authors: | , , , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Public Library of Science (PLoS)
2020-01-01
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Series: | PLoS ONE |
Online Access: | https://doi.org/10.1371/journal.pone.0244215 |