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Article

Bioengineered AAV9 and Optimised Microdystrophin Vectors Augment Phenotypic Rescue in a Murine Model of Duchenne Muscular Dystrophy.

Journal of cellular and molecular medicine - 1 Mar 2026

Senthilkumar Mohankumar B, Sharma Sanya, Srinivasan Navaneeth, Varghese Anila, Sarangi Pratiksha, Singh Vijayata, Kumar Narendra, Yenurkar Devyani, Mukherjee Sudip, Amit Sonal, Bhatia Sameer, Misra Santosh K, Puri Ratna Dua, Chamberlain Jeffrey, Jayandharan Giridhara R

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder without an effective cure. Adeno-associated virus (AAV) based gene therapy has improved dystrophin function, with sub-optimal clinical outcomes. We reasoned that a combination of rational engineering of AAV9 capsids modified at the post-translational modification sites, optimal promoter selection, and codon-optimisation of the microdystrophin...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 41793215
DOI: 10.1111/jcmm.71078
PMCID: PMC12966934

Genes

DMD

Provenance

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

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