The Japanese Biochemical Society

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[2T11m-03(2P-043)] AAV-mediated gene therapy for GM2 gangliosidosis model mouse

Ohnishi Yukiya1, Tsuji Daisuke1,2,4, Watanabe Ryosuke2, Muramatsu Shinichi3,4,5, Koji Itoh1,2,4,5 (1.Dept. of Medicin. Biotechnol. , Grad. Sch. of Biomed. Sci. , Tokushima Univ., 2.Facult. of Pharmaceut. Sci. , Tokushima Univ., 3.Facult. of Med. , Jichi Med. Univ., 4.AMED, ACT-MS, 5.AMED, TR-SPRINT)

キーテクノロジー:遺伝子治療

GM2ガングリオシド、β-Hexosaminidase、リソソーム病、遺伝子治療、アデノ随伴ウイルス

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