Article

Improving angiogenesis ameliorates the efficacy of ASO-based exon-skipping for the treatment of Duchenne muscular dystrophy

2025-08-21

Abstract excerpt

<h4>ABSTRACT</h4> Duchenne muscular dystrophy (DMD) is a severe X-linked disease caused by mutations in the DMD gene, resulting in the absence of functional dystrophin. Antisense oligonucleotide (ASO)-based therapies aim to restore the open reading frame and produce a truncated but functional dystrophin protein. Although several ASOs are FDA-approved, dystrophin restoration in patient biopsies remains low, under...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: aa60634d-3f19-5e36-9fa2-053d41bbb8c6
DOI: 10.1101/2025.08.21.670323

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Improving angiogenesis ameliorates the efficacy of ASO-based exon-skipping for the treatment of Duchenne muscular dystrophyDOI 10.1101/2025.08.21.670323Read

Next steps for the optimization of exon therapy for Duchenne muscular dystrophySemantic proximity Read

Antisense oligonucleotide-mediated exon skipping for Duchenne muscular dystrophy: progress and challenges.Semantic proximity Read

Phase I/II Trial of Brogidirsen: Dual-Targeting Antisense Oligonucleotides for Exon 44 Skipping in Duchenne Muscular DystrophySemantic proximity Read

Brogidirsen and Exon 44 Skipping for Duchenne Muscular Dystrophy: Advances and Challenges in RNA-Based TherapySemantic proximity Read

Systemic Delivery of Human Mesangioblasts mediated by a Nanofiber Scaffold restores Dystrophin Expression in Immunodeficient Dystrophic MiceSemantic proximity Read

<i>DMD</i> antisense oligonucleotide mediated exon skipping efficiency is affected by flanking intron retention time and target position within the exonSemantic proximity Read

Overview on DMD exon skipping.Semantic proximity Read

RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying StrategiesSemantic proximity Read

RNA Therapeutics for Duchenne Muscular Dystrophy: Exon Skipping, RNA Editing, and Translational Insights from Genome-Edited Microminipig Models.Semantic proximity Read

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseasesSemantic proximity Read

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides.Semantic proximity Read

The Future of Exon Skipping for Duchenne Muscular Dystrophy.Semantic proximity Read