
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.
Mice, Knockout, Gangliosidosis, GM1, Genetic Therapy, beta-Galactosidase, β-ガラクトシダーゼ, Disease Models, Animal, Mice, 遺伝子治療, Animals, Humans, ノックアウトマウス, G<SUB>M1</SUB>-ガングリオシドーシス
Mice, Knockout, Gangliosidosis, GM1, Genetic Therapy, beta-Galactosidase, β-ガラクトシダーゼ, Disease Models, Animal, Mice, 遺伝子治療, Animals, Humans, ノックアウトマウス, G<SUB>M1</SUB>-ガングリオシドーシス
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