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

Expression of full-length dystrophin reverses muscular dystrophy defects in young and old mdx4cv mice

10 Jun 2025

Abstract excerpt

Gene replacement therapies mediated by adeno-associated viral (AAV) vectors represent a promising approach for treating genetic diseases. However, their modest packaging capacity (~4.7 kb) remains an important constraint and significantly limits their application for genetic disorders involving large genes. A prominent example is Duchenne muscular dystrophy (DMD), whose protein product dystrophin is generated...

Read the complete abstract on PubMed

Topics

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

Share to Adipose Tissue and Metabolism
Share to Muscle Physiology and Disorders
Share to Virus-based gene therapy research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 40493400
DOI: 10.1172/jci189075

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Expression of full-length dystrophin reverses muscular dystrophy defects in young and old mdx4cv micePMID 40493400Read ↗

Split intein-mediated protein trans-splicing to express large dystrophinsSemantic proximity Read ↗

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors.Semantic proximity Read ↗

In Silico Structural Prediction for the Generation of Novel Performant Midi-Dystrophins Based on Intein-Mediated Dual AAV ApproachSemantic proximity Read ↗

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy MuscleSemantic proximity Read ↗

Lentiviral vectors can be used for full-length dystrophin gene therapySemantic proximity Read ↗

Lentiviral vectors can be used for full-length dystrophin gene therapySemantic proximity Read ↗

Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors.Semantic proximity Read ↗

In Silico Structural Prediction for the Generation of Novel Performant Midi-Dystrophins Based on Intein-Mediated Dual AAV ApproachSemantic proximity Read ↗

AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospectsSemantic proximity Read ↗

The DMD gene and therapeutic approaches to restore dystrophin.Semantic proximity Read ↗

Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy.Semantic proximity Read ↗

Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMDSemantic proximity Read ↗

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