Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

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

International journal of molecular sciences - 18 Mar 2026

Chassin Alex, Ono Hiroya, Ashida Yuki, Imamura Michihiro, Aoki Yoshitsugu

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular disease (NMD) caused by loss-of-function mutations in the DMD gene. RNA-based therapies, especially antisense oligonucleotides (ASO)-mediated exon skipping and adenosine deaminase acting on RNA (ADAR)-guided RNA editing, have emerged as complementary approaches that modulate pre-mRNA splicing or correct transcripts without altering genomic DNA....

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Dystrophin
Share to Exons

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Share to Gene Editing

Share to Genetic Therapy

Share to Morpholinos

Share to Muscular Dystrophy, Duchenne

Share to Oligonucleotides, Antisense

Share to RNA Editing

Source

Open in PubMed Open full text

Identifiers

PMID: 41898615
DOI: 10.3390/ijms27062755
PMCID: PMC13027266

Genes

DMD
ADAR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 23 Oct 2026.

Related research

Semantic proximity does not establish scientific evidence. Graph snapshot: 2026-09-29.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

RNA Therapeutics for Duchenne Muscular Dystrophy: Exon Skipping, RNA Editing, and Translational Insights from Genome-Edited Microminipig Models.PMID 41898615Read ↗

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

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

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

Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing.Semantic proximity Read ↗

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

Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMOSemantic proximity Read ↗

Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophySemantic proximity Read ↗

Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse modelSemantic proximity Read ↗

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

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

Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translation.Semantic proximity Read ↗

PPMO-mediated exon skipping induces uniform sarcolemmal dystrophin rescue with dose-dependent restoration of circulating microRNA biomarkers and muscle biophysical propertiesSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.