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Efficacy and muscle safety assessment of fukutin-related protein gene therapy

Halli Benasutti; Joseph W. Maricelli; Jane Seto dkk.

Molecular Therapy: Methods & Clinical Development, Vol. 30 · 2023 · Inggris · DOAJ

Limb-girdle muscular dystrophy type R9 (LGMDR9) is a muscle-wasting disease that begins in the hip and shoulder regions of the body. This disease is caused by mutations in fukutin-related protein (FKRP), a glycosyltransferase critical for maintaining muscle cell integrity. Here we investigated…