[GM1-gangliosidosis knockout mouse].
We generated a beta-galactosidosis mouse by gene targeting in an embryonic stem cell. Clinical, pathological, and biochemical analyses revealed that this mouse is a useful animal model to study the pathogenesis and therapy of human GM1-gangliosidosis.
Oshima
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Department of Veterinary Science, National Institute of Infectious Disease, Tokyo.
D000818:Animals; D004195:Disease Models, Animal; D016537:Gangliosidosis, GM1 / Q000209:etiology / Q000473:pathology / Q000628:therapy; D015316:Genetic Therapy; D006801:Humans; D051379:Mice; D018345:Mice, Knockout; D001616:beta-Galactosidase / Q000235:genetics
D001616:beta-Galactosidase