liposomal GNE gene therapy

Liposomal GNE gene therapy is an investigational gene replacement therapy designed to treat GNE myopathy, a rare autosomal recessive muscle-wasting disorder. The therapy utilizes a non-viral liposomal delivery system to transport a functional copy of the *GNE* gene, which encodes the bifunctional enzyme UDP-GlcNAc 2-epimerase/N-acetylmannosamine kinase. This enzyme is critical for the biosynthesis of sialic acid; mutations in the *GNE* gene lead to hyposialylation of muscle proteins, resulting in progressive muscle atrophy. By delivering a wild-type *GNE* plasmid, the therapy aims to restore enzyme activity and normalize sialic acid production in muscle tissues, potentially halting or reversing disease progression. Developed by researchers at the Manipal Academy of Higher Education (MAHE) in collaboration with the HIBM Research Group, it represents one of the first gene therapies to enter clinical trials in India.

Other names

GNE gene therapyLiposomal-mediated GNE gene therapyGNE-liposome complex