How Is Exon Skipping Therapy Redefining DMD Treatment

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2026-05-13 09:12:05

Exon skipping therapy for Duchenne muscular dystrophy — the antisense oligonucleotide (ASO) approach using synthetic nucleic acid molecules that bind pre-mRNA splicing signals to cause targeted exon exclusion during RNA processing, converting the out-of-frame DMD gene mutations causing Duchenne muscular dystrophy into in-frame mutations producing a shorter but partially functional...

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