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Article

Therapeutic potential of highly functional codon-optimized microutrophin for muscle-specific expression.

Scientific reports - 17 Jan 2022

Starikova Anna V, Skopenkova Victoria V, Polikarpova Anna V, Reshetov Denis A, Vassilieva Svetlana G, Velyaev Oleg A, Shmidt Anna A, Savchenko Irina M, Soldatov Vladislav O, Egorova Tatiana V, Bardina Maryana V

Abstract excerpt

High expectations have been set on gene therapy with an AAV-delivered shortened version of dystrophin (µDys) for Duchenne muscular dystrophy (DMD), with several drug candidates currently undergoing clinical trials. Safety concerns with this therapeutic approach include the immune response to introduced dystrophin antigens observed in some DMD patients. Recent reports highlighted microutrophin (µUtrn) as a less...

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Identifiers

PMID: 35039573
DOI: 10.1038/s41598-022-04892-x
PMCID: PMC8764061

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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