Methods in molecular biology (Clifton, N.J.)AnimalsMiceExonsMuscular DystrophyDuchenne
Splice Modulation Studies Using Reporter Mice.
Katsura Minegishi, Tsukasa Tominari, Yoshitsugu Aoki
Published: 202610.1007/978-1-0716-4738-7_9
Abstract
Exon-skipping therapy for Duchenne muscular dystrophy (DMD) recovers muscle function by inducing shorter but functional dystrophin protein using antisense oligonucleotides (ASOs) that modulate the pre-mRNA splicing of DMD transcripts to restore the d…
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