Abstract
Feline ganglioside storage diseases are authentic models of the human conditions and are valuable for development of gene therapy strategies for human patients. Deficiency of the lysosomal enzymes β-galactosidase or β-N-acetylhexosaminidase causes continuous cumulation of GM1 or GM2 ganglioside, respectively, resulting in disease manifestations nd death. There is no reliable treatment for human gangliosidoses, although knockout mouse models have been used to develop promising experimental treatments including gene therapy with adeno-associated virus vectors.
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