Evaluation of N-nonyl-deoxygalactonojirimycin as a pharmacological chaperone for human GM1 gangliosidosis leads to identification of a feline model suitable for testing enzyme enhancement therapy

► We identify NN-DGJ as a pharmacological chaperone for human GM1 gangliosidosis. ► NN-DGJ enhances residual activity in cells with 3 out of 7 missense mutations tested. ► NN-DGJ acts as a pharmacological chaperone in cells from a feline model of GM1. ► This GM1 feline model is the first suitable for testing chaperone therapy in vivo.