Article

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

2024-08-30

Abstract excerpt

<h4>Summary</h4> Duchenne muscular dystrophy (DMD) is a severe muscle disorder caused by mutations in the DMD gene, resulting in dystrophin loss. Exon-skipping using antisense oligonucleotides (ASO) is a promising approach that partially restores dystrophin by correcting the frameshift during pre-mRNA splicing. However, a weakness of the current approach is that it is mutation-specific and has poor efficacy. To ad...

Topics

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

Identifiers and source

Literature Corpus work: 9d50e140-f15a-5654-acf5-35e91c86475e
DOI: 10.1101/2024.08.28.24312624

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Phase I/II Trial of Brogidirsen: Dual-Targeting Antisense Oligonucleotides for Exon 44 Skipping in Duchenne Muscular DystrophyDOI 10.1101/2024.08.28.24312624Read

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

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

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

Systemic administration of the antisense oligonucleotide NS-089/NCNP-02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: study protocol for a phase I/II clinical trialSemantic proximity Read

AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophySemantic proximity Read

Recent Trends in Antisense Therapies for Duchenne muscular dystrophySemantic proximity Read

Systemic administration of the antisense oligonucleotide NS-089/NCNP-02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: Study protocol for a phase I/II clinical trial.Semantic proximity Read

Emerging Oligonucleotide Therapeutics for Rare Neuromuscular DiseasesSemantic proximity Read

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

Clinical trials of exon skipping in Duchenne muscular dystrophySemantic proximity Read

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

Exon skipping: a first in class strategy for Duchenne muscular dystrophySemantic proximity Read