Preclinical assessment of GNEwt/bi-shRNA-GNEM743T lipoplex product development for GNE myopathy

Aims GNE myopathy is a heredity disease of unmet medical need associated with progressive skeletal muscle wasting, atrophy and weakness caused by mutations in the GNE gene. GNE plays a pivotal role in sialic acid production. Sialic acid is a critical part of glycoprotein, ganglioside and glycolipid...

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Hlavní autoři:	Fabienne Kerneis, Christopher M Jay, Donald Rao, Jeremy Winchester, Alexander Nemunaitis, Emily Nemunaitis, Ernest Bognar, Gladice Wallraven, Laura Stanbery, Adam Walter, Sep Sarshar, John Nemunaitis
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Taylor & Francis Group 2026-12-01
Edice:	Future Science OA
Témata:	Plasmid bi-shRNA GNE myopathy GNE M743T
On-line přístup:	https://www.tandfonline.com/doi/10.1080/20565623.2026.2635722
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